For cancer treatment and more, genetic-based precision medicine holds a lot of promise – Connecticut Magazine

A month following surgery for thyroid cancer, a Hartford Hospital patients tumor grew to 10 inches. The case was presented to the hospitals tumor board, which involved 30 doctors from different specialties.

The gene mutation found to be controlling the patients tumor growth was already well-established as a driver of melanoma, the deadliest form of skin cancer, says Dr. Sope Olugbile, medical oncologist at Hartford HealthCare.Chemotherapy wouldnt work fast enough against the aggressive tumor. Tumor board members recommended a targeted therapy already treating patients with melanoma. Without that genetic information, we wouldnt have been able to come up with that therapy, he says. The treatment saved the patients life, so far. Our goal is to use more of the genetic information to drive the treatment of cancer patients.

This type of personalized care, known as precision medicine and its subset, genomic medicine, has been offered for years at world-renowned cancer-treatment hospitals such as Memorial Sloan Kettering Cancer Center in New York, Dana-Farber Cancer Institute in Boston and University of Texas MD Anderson Cancer Center in Houston. Its now the standard of care in Connecticuts Hartford HealthCare Cancer Institute, UConn Health Center in Farmington, Connecticut Childrens Medical Center in Hartford and Smilow Cancer Center at Yale New Haven Health.Cancer therapy has become precision therapy, says Dr. Roy Herbst, professor of medicinal oncology and pharmacology, and chief of medical oncology at Yale Cancer Center and Smilow Cancer Hospital.

Dr. Roy Herbst, of Yale Cancer Center and Smilow Cancer Hospital, says that precision care is often used in cancer treatment these days.

While its most commonly used with cancer patients, precision medicine is also making inroads into other areas of health care including the treatment of some cardiac patients. Its also being studied and used on a limited basis to treat those with rare diseases. In the U.S., newborns are screened with a blood test for hearing loss and heart defects. If detected and treated early, this can prevent death and disability in some cases. For some doctors and researchers, precision medicine holds the promise of effective targeted diseases and chronic conditions, and, even more revolutionary, the chance to prevent illness before it arises. The race is on to gather as much data as possible in order to increase understanding of the connection between genes and overall health; here in Connecticut, Yales Center for Genetic Health last fall launched its Generations project to collect DNA from 100,000 volunteers (see sidebar below).

Precision medicine involves the study of human genes, called the genome. The human genome contains 23 pairs of chromosomes within all human cells, and each chromosome contains hundreds to thousands of genes. Using high-level computing and mathematics, genomics researchers analyze massive amounts of DNA-sequence data to find variations or mutations that affect health, disease or response to drugs, according to an online description by The Jackson Laboratory for Genomic Medicine in Farmington.

Researchers can sequence an entire tumor to look for markers or abnormalities that can be treated with a targeted medication that attacks that mutation, unlike traditional chemotherapy that kills healthy cells along with cancer cells, says Herbst, also associate director for translational science at the Yale School of Medicine.

These days, when Yales precision medicine tumor board meets weekly, they dont focus on where the tumor began, he says. They look at what errors occurred in the DNA of the tumor, because once they know whats driving the tumor, they can treat it.For example, lung cancer is the most common cancer in the world. When a nonsmoker gets lung cancer, doctors sequence the tumors DNA to see if it contains one of eight genes known to mutate.

Each cancer cell has about 18,000 to 20,000 genes, and there are some cancers where just one of those genes is directing the growth of the cancer, Olugbile says. We call that the driver gene. The other 17,999 are just following the lead of that driver gene, he says. That means if we tag just that one gene with the medication then we can actually shut down the growth of the entire cancer.

Traditional chemotherapy can only be given for 4-6 months because of the side effects, while targeted oral medications have very few side effects and patients remain on them for an average of two years, Olugbile says.

In the past five years, genetic testing has become standard of care for some cancers specifically colon, lung and melanoma because those types of cancers tend to have genetic mutations that have been known to respond to therapy, says Sara Patterson, manager of clinical analytics and curation at Jackson Labs, which works with UConn and Yale researchers.But targeted therapy is not a cure-all, and researchers are still a long way from using precision medicine to treat all cancer patients. Even if cancers have the same genomic change and mutation, theres no guarantee they will all respond to the same therapy, she says.Overall, precision medicine is only effective at stopping the spread of cancer in an average of 20 percent of cancer patients treated, Olugbile says, with variations by cancer. Sometimes the cancer returns because the tumor changes to resist the therapy, Patterson adds.

As doctors and researchers do more genomic sequencing, the data pool will grow and so will knowledge of what medications work most effectively against various tumor types.The more information we gather, the better well know how to treat specific patients, Patterson says.

Reimbursement from insurance companies can be a challenge. If precision treatment for a particular type of cancer hasnt been approved by the insurance industry, its difficult to get reimbursed for genomic testing, says Sue Mockus, director of product innovation and strategic commercialization at Jackson Labs.Its a catch-22. Even though a patient with pancreatic cancer could benefit from a targeted therapy, unless that patient is part of a clinical trial that would pay for the genomic testing, the patient would have to pay out of pocket, the annual cost of which can run into the hundreds of thousands of dollars. If you do have a mutation identified and your physician wants to give you the medication off label, you have to fight with the insurance company, Mockus says.

Experts have suggested a value-based approach to precision medicine, reports the International Journal of Public Health. This means policy decisions about reimbursement and investment in research and development will factor in how long patients lives are prolonged and the quality of those lives, the Journal reports.

Oncologists also offer cancer patients immunotherapy, another form of personalized medicine, Patterson says. Theyre using diagnostic tests on tumors, independent of genomic sequencing, to determine if their tumor profiles make them a good immunotherapy candidate. Immunotherapy is approved for multiple tumor types, as long as they have certain markers, she says.

Former President Jimmy Carter became cancer free after receiving radiation and immunotherapy to treat the melanoma that had spread to his brain and liver. While immunotherapy can cure cancer for some, its only effective about 20 percent of the time, Olugbile says. It varies a bit by cancer, with some cancers having a higher success rate, he adds.

Through a collaboration with Memorial Sloan Kettering, Hartford HealthCares Advanced Disease Clinic was scheduled to open this spring to give patients even more options, he says. If targeted therapies and immunotherapies dont work or are not a match for patients, doctors will look for suitable clinical trials that offer potential treatments, Olugbile says.Our goal is to create awareness on two fronts, one is among the doctors. Yes, we are available to help if patients have gone through standard of care who didnt respond, he says. Its also an option for patients who want to be treated with precision medicine closer to home. The goal is to make it available so they dont have to go to New York or Boston, he says. Its right here in Hartford and hopefully at other cancer centers over time.

From Yale, Herbst leads a clinical trial through the National Cancer Institute where he and his team are trying to match the right patient to the right drug.Every tumor is getting sequenced. Thats accelerating the field. The sequencing techniques have gotten cheaper and faster, so we can analyze them at the point of care, Herbst says. This is why clinical trials are so important. Whats a clinical trial today is standard of care tomorrow.

In a study published in the journal Science Translational Medicine, a multi-institutional research team including a Connecticut doctor developed an advanced method to analyze existing data from thousands of clinical trials, comparing which genes FDA-approved drugs work against to the genes active in pediatric brain tumor patients. This sped up the lengthy process of developing cancer drugs.

Dr. Ching Lau, head of the oncology-hematology division at Connecticut Childrens Medical Center and the pediatric oncology-hematology department at UConn School of Medicine, is accessing the World Community Grid, an IBM-funded program that allows researchers worldwide to perform tens of thousands of virtual experiments. Instead of screening thousands and thousands of compounds to try to find a potential drug, we found we could use genomics data already available and do a more systems-approach analysis to figure out the predominant pathways driving the tumor cells, Lau, professor at The Jackson Laboratory, says in an email. Then we asked if there were any existing FDA-approved drugs that could potentially modulate those pathways.

The researchers identified eight drugs that could potentially fight medulloblastoma (MB) tumors, the most common malignant brain tumor in children. One of the drugs showed an increased survival rate in mice with MB tumors, and a clinical trial is being pursued.

Personalized medicineand heart disease

Precision medicines applications have expanded beyond cancer care. At first, much heart disease research relied on a genetic analysis of whether someone was predisposed to a disease. Thanks to a growing database of patient information that is shared worldwide, researchers can mine huge data sets with hundreds of thousands of cases for patterns and abnormalities that lead to discoveries, says Beth Taylor, associate professor of kinesiology at UConn and director of exercise physiology research in cardiology at Hartford Hospital. Researchers and clinicians know that about half the people who have heart attacks dont have the typical risk factors such as high blood pressure, obesity and diabetes. To determine why physically active people with healthy diets have heart attacks, researchers are using precision medicine to comb through large studies to find small predictors, Taylor says. Often the influence of any one factor is hard to detect unless you have a big sample size, she says.

The National Institutes of Health requires grant recipients to share their data to a national registry so that researchers have access to big data, she says. (Personal information such as date of birth, name and address are removed from files used for research studies.)

When we first began to really measure genetic variations, it was believed that was going to be the big hope in treatment, Taylor says. But genes are complex and environmental factors modify genetics for multiple generations.

For the first time ever, weve got wide-scale computing ability to analyze huge data points. This can better allow us to predict disease progression and optimize treatment, she says. Many of us would say that this concept of big data is as or more important than genetic risk. Genetic risks are not the whole picture.

For the first time ever, weve got wide-scale computing ability to analyze huge data points. This can better allow us to predict disease progression and optimize treatment.

Progress with diabetes

Precision medicine is not widely used in the treatment ofdiabetesin the U.S., except when it comes to a rare form of diabetes called neonatal diabetes mellitus. While type 1 and type 2 diabetes are controlled by two or more genesand additional genetic factors,neonatal diabetes mellitus involves a single gene and develops in babies under 6 months old.

Through genetic testing of babies with elevated blood sugar levels,researchers learnedthat about half the patients have gene mutations that respond well to a pill used to treat type 2 diabetes and they dont need to be on insulin for the rest of their lives like type 1 diabetics, says Karel Erion,director of research stewardship and communications for the American Diabetes Association.

When infants show signs of type 1 diabetes at Yale New Haven Childrens Hospital or Connecticut Childrens Medical Center, they are automatically tested for neonatal diabetes, hospital doctors say.

An example of precision medicine as a predictor of disease is the TrialNet database, which uses genetic testing to determine whether the relatives of those with type 1 diabetes have two or more of the five diabetes-related autoantibodies (proteins produced by the immune system directed against the persons own proteins) linked to increased risk of developing type 1 diabetes. Type 1 diabetics must take insulin for the rest of their lives to survive, and theres no known way to prevent the autoimmune disease. Type 1 diabetes, formerly called juvenile diabetes, typically strikes children and adolescents, causing the pancreas to stop producing insulin, a hormone needed to process sugar, or glucose, from food. Type 2 diabetes was formerly known as adult-onset diabetes, but the disorder is being seen in more children, thought to be the result of a rise in childhood obesity. Screening identifies the early stages of the disease years before any symptoms appear, according to the TrialNet website.

In a study published in the New England Journal of Medicine, researchers from the TrialNet Study Group, led by Yale Universitys Dr. Kevan Herold, found that an experimental medication delayed the onset of type 1 diabetes in high-risk participants by two years compared to the control group. The disease was diagnosed in 43 percent of the participants who received the medication, teplizumab, and 72 percent of those who received the placebo.

Alzheimers disease and dementia

Only 1 to 3 percent of the 5 million people living with Alzheimers disease have a genetic mutation that leads to whats called genetic or familial Alzheimers. But one in three older adults will eventually develop some form of dementia, says Rebecca Edelmayer, the Alzheimers Association director of scientific engagement.

Like other diseases that strike large segments of the population, researchers rely on big data to learn about Alzheimers and which genes play a role in who gets it.Researchers have learned that there are several risk factors that contribute to dementia, she says. Specifically, the presence of heart disease, high blood pressure, diabetes, social and cognitive isolation, poor nutrition and the level of education, can contribute to cognitive decline, she says.

Scientists from around the world share research data and draw from data in the Global Alzheimers Association Interactive Network, she says.The field has made some dramatic advances in understanding of how genetics play a role and how other underlying diseases play a role, Edelmayer says. We need to give doctors evidence-based recommendations.

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For cancer treatment and more, genetic-based precision medicine holds a lot of promise - Connecticut Magazine

Drug factories: GMOs and gene editing are poised to transform medicine. Here’s how. – Genetic Literacy Project

No one likes getting a shot at the doctors office. As kids, we werent used to having a sharp needle prick our skin, let alone by someone doing it on purpose. An estimated 10% of the population is affected by trypanophobia the fear of needles or injections. Luckily, for most, shots are an infrequent occurrence often limited to vaccinations. However, for millions of others, injections are a more frequent fact of life required in dealing with disease. The need for these injections and their associated doctor visits mean the physical discomfort of the treatment is often compounded by a financial burden.

Fortunately, plant biotechnology is poised to drastically improve how we consume medication. Using the modern tools of genetic engineering, researchers are developing plant-based drugs that are cheaper, easier to take and even more effective than their existing counterparts.

Cant more medicines be reformulated for oral delivery?

While many diseases can be treated with orally administered medications, other drugs such as biologics or biopharmaceuticals, medicines derived from living organisms, must be delivered using other strategies. Conventional drugs like aspirin are chemically synthesized and can survive digestion, whereas biologics like hormones, antibodies, enzymes, and other complex organic molecules are vulnerable to degradation by enzymes in our saliva and stomach, as well as environmental conditions like pH and heat. This makes biologics in pill form unlikely to survive the harsh environment of the digestive tract.

Pricey biologics

In addition to the unpleasant nature of biologic injections is their associated costs. Biologics are made by taking the DNA blueprint for the molecule and expressing it in bacterial, yeast, or mammalian cells. Once these cells, typically grown in large vats filled with nutrient media, produce the molecule of interest, it must be isolated and purified. Each step of this process must be exact and carefully maintained as small variations may change the structure and identity of the drug, potentially altering its behavior. This complex manufacturing process in addition to more rigorous FDA regulations mean higher drug prices for consumers. Combined with the price of doctor visits to get these frequent injections or infusions, the annual cost of some biologics can reach hundreds of thousands of dollars.

There are more than 200 FDA-approved biologic drugs. While less than two percent of people in the US rely on biologics, they make up 40 percent of prescription drug spending. Identifying a better way to produce and administer biologics has the potential to ease the physical and financial burden associated with these drugs. For this reason, researchers are turning to the original inspiration for medications: plants.

Turning plants into pharmaceutical factories

Evidence for plant use in medicine dates back all the way to the Palaeolithic Age. But instead of trying to find new plants that produce medically relevant compounds, researchers are turning to genetic engineering to express the same biologics currently grown in bacterial, yeast, or mammalian cells.

Producing biologics in plants has a number of advantages. Plants are potentially less costly to grow, requiring inexpensive fertilizers instead of specialized cell culture growth media. Plants can also be grown in fields or greenhouses without requiring sterile environments, meaning that scaling up production would just require more growing area as opposed to additional expensive bioreactors. An added benefit is that plants do not serve as hosts for human pathogens, reducing the likelihood of harm from contaminants that bacterial or mammalian cells may house.

Once the drug-producing plants are grown, the medically relevant proteins may be extracted and purified. But plants allow for this platform to be taken one step further: by turning the biologic- expressing plants into a freeze-dried (lyophilized) powder and placing it into a capsule, the drugs can be delivered orally. Plant cell walls contain cellulose which cannot be digested by enzymes in the stomach but can be broken down by the commensal bacteria living in our intestines. Plant-encapsulated drugs are then released in the blood-rich absorptive environment of the small intestine, where they become bioavailable and distributed to target tissues. By producing these drugs in a lyophilized form, manufacturers can cut out the expensive purification process and the need for cold transport and storage.

Current research efforts

Theres been some reported success using this method, including a March 2020 paper from a team at the University of Pennsylvania describing a lettuce expressing a novel human insulin-like growth factor-1 (IGF-1). IGF-1 helps promote skeletal muscle and bone development. For this reason, IGF-1 injections have been used in the treatment of several muscle disorders and have the potential for therapeutic benefit in healing bone fractures.

To study if plant-grown IGF-1 might be an effective replacement for traditional IGF injections, the team modified human IGF-1 to allow for uptake through the gut. They found that their modified version not only stimulated proliferation of several cell types better than current commercial IGF-1, but also that the plant-encapsulated drug could be administered orally to mice and would effectively be delivered to blood serum. The team also found that this administration of the drug significantly increased bone density in diabetic mice as compared to a control group.

In addition to medication production, companies are also looking to utilize some of the benefits of plant-based production for vaccines. Medicago, a Canada-based company seeking approval for their plant-produced flu vaccine, has announced that using this same technology, they have produced a candidate vaccine for COVID-19 in twenty days. By growing the protein for the vaccine in plants, as opposed to using eggs to propagate the virus, Medicago has been able to cut the cost and time required to produce a new vaccine. The vaccine is now awaiting clinical testing and FDA approval.

Similar to the work on orally administered IGF-1, theres also a lot of interest in making edible vaccines. In the future, you may no longer need to go to a clinic to get a seasonal flu vaccine, but instead eat a salad made with vaccine-containing lettuce or tomatoes. This could potentially reduce patient discomfort and increase vaccine compliance, minimizing everybodys risk of contracting infectious diseases. Edible vaccines would also help expand access to immunization in parts of the world were delivering vaccines may be difficult.

Plant-produced pharmaceuticals have the potential to improve the quality of life for millions of people by reducing the physical and financial burden of relying on biologics to stay healthy. There may even come a day when getting a shot at the doctors office is a thing of the past replaced by a quick trip to the grocery store.

Tautvydas Shuipys is a PhD candidate in the Genetics and Genomics Graduate Program at the University of Florida. Follow him on Twitter @tshuipys

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Drug factories: GMOs and gene editing are poised to transform medicine. Here's how. - Genetic Literacy Project

How to know your risk factors for hypertension and whether high blood pressure is genetic – Insider – INSIDER

Hypertension is the medical term for high blood pressure. It can cause serious health problems, like heart disease or stroke, if left untreated.

There are many risk factors that make it more likely for someone to develop hypertension. These include genetic factors, age, race, medical conditions, and unhealthy lifestyle choices.

Here's what you need to know about what increases the risk of high blood pressure.

Overall, one or more of these factors are most likely to cause high blood pressure:

One of the biggest culprits is smoking, which can double or triple the risk of developing hypertension. That's because smoking damages blood vessels and can reduce blood flow to the heart.

But these factors won't always cause hypertension alone genetics and medical conditions can also increase your risk.

"I would say the simplest answer to this is, yes, there is a genetic component," says Joshua Shatzkes, MD, a cardiologist at Mt. Sinai Hospital.

If your parents have hypertension, you are at an increased risk for high blood pressure.

A 2018 study involving over a million people identified 500 genes that influence blood pressure. Some of these genes influence the cells lining blood vessels, causing them to be abnormally constricted and raise blood pressure.

Other genes can cause high cholesterol, especially in a condition known as hypercholesterolemia, which can also increase blood pressure.

In addition, genetic factors often combine with other adverse lifestyle choices, which can further increase the risk of hypertension.

For example, when you're growing up, if your family eats an unhealthy diet high in sodium, creates a high stress environment, smokes too many cigarettes, and doesn't exercise often, then you're more likely to inherit those behaviors and more likely to develop hypertension.

While there's no universal way to describe obesity, it's often considered a condition where someone has a high body mass index (BMI). A BMI over 40 can double or triple your likelihood of developing obesity-related illnesses.

"When someone is obese, it simply takes more work for the heart to pump blood throughout the whole body," says Christopher Granger, MD, a cardiologist at Duke Health. "And when it has to pump blood throughout the whole body, it has to generate a higher kind of pressure to do so."

In fact, a study from 2015 suggests that excess body fat accounts for 65% to 75% of hypertension cases. Moreover, a 2017 study found that childhood obesity increased the rate of developing adult hypertension by 65%.

Diabetes is a condition where your body doesn't react to high blood sugar properly, which may cause it to be too high or too low longer than normal. Insulin is the hormone designed to take glucose (sugar) from the blood to the cells, but if there's not enough insulin, glucose stays in the blood, elevating blood sugar levels.

Over time, high blood sugar can cause plaque to build up in your blood vessels, which narrows the vessel and increases blood pressure.

Granger explains that high blood sugar can cause the arteries to stiffen because it can increase the production of free radicals tiny particles that damage cells and reduce nitrous oxide, a chemical that dilates blood vessels.

As a result, 30% of people with type 1 diabetes develop hypertension. Those with type 2 diabetes are 2.5 times more likely to develop hypertension and 50% to 80% will develop hypertension. However, diabetics who carefully control their blood sugar levels can effectively decrease the risk of developing hypertension.

The American Heart Association has found that over 40% of non-Hispanic African-Americans have high blood pressure, and that it can develop earlier in their lives and become more severe.

Overall, black Americans are twice as likely to develop hypertension by the age of 55 compared to white Americans. Systemic issues could explain this increase in blood pressure.

There's an association between racism and higher blood pressure in African-American men, according to the CDC. Black Americans are also exposed to more factors that can increase chronic stress such as discrimination and lower socioeconomic status which may contribute to high blood pressure.

Older people are more likely to develop hypertension because the arteries stiffen as we age. This process is called atherosclerosis, and it describes plaque build-up in blood vessels.

According to Granger, young people's arteries are able to expand and more effectively accommodate the pulse of blood flow.

Overall, your lifetime risk for developing hypertension is 90%, according to Johns Hopkins Medicine. Even if you have heart-healthy habits, you'll still most likely develop hypertension, according to the National Institute on Aging.

However, certain lifestyle changes, such as exercising every day, sleeping adequately, and avoiding smoking, can lower the risk of developing high blood pressure.

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How to know your risk factors for hypertension and whether high blood pressure is genetic - Insider - INSIDER

Venture capital found its footing in biotech. Then came the virus. – BioPharma Dive

Amir Nashat has spent nearly two decades building biotechnology companies. The first he worked on, Alnylam Pharmaceuticals, pioneered a new way to make genetic medicine. He's since helped advise and nurture at least 16 others, several of which were acquired for hundreds of millions of dollars.

Despite this track record, Nashat, a partner at the venture firm Polaris Partners, says most of his career in venture capital took place under "really crappy circumstances" that made it challenging to invest in young drug companies. Only in the last five or so years did things really start to change.

It was during this period that public markets came to love young biotechs, buying into record stock offerings. Large drugmakers, starved for innovation, also turned to them for their next drugs. This created a "hyper-compressed, hyper-intense environment," according to Nashat, where venture firms had much clearer and quicker paths to earn returns on their investments. For venture capitalists, there had never been a better time to invest in drug startups and, coming into 2020, many expected another big year.

Their predictions were quickly upended by the spread of the new coronavirus, which has infected millions and brought the global economy to a halt. In the past, economic downturns shaped how venture firms fund and incubate drug companies. Now, a pandemic threatens to do the same.

BioPharma Dive spoke with half a dozen venture capitalists who grow drug companies, as well as legal and financial advisors who work with healthcare venture firms. Almost all said the spread of the new coronavirus is affecting to some degree how they manage existing investments or think about new ones.

"It used to be that we had a lot of chaos, but the rest of the world was predictable," said Noubar Afeyan, CEO of Flagship Pioneering, the biotech incubator which founded coronavirus vaccine developer Moderna along with more than 25 other companies. "Now, we have chaos and the rest of the world has chaos, and so there are some adjustments being done."

As venture capitalists assess the damage caused by the pandemic, they appear to be treading lightly financial data provider PitchBook found biopharma venture deals are down roughly 16% compared to last year. Some firms told BioPharma Dive that, in the current environment, they'd be apprehensive to invest in certain kinds of drug companies.

Even small adjustments could have a lasting impact on the drug industry, given the vital role venture firms play in it. Many biotechs wouldn't exist without venture money and support, making these investors a powerful force over the drugs that could become available in the future.

After the recession in the early 2000s, scientific breakthroughs led to a surge in biotech investments, many of which would ultimately disappoint. When the financial crisis hit in 2008, healthcare-focused venture firms found it extremely difficult to raise money from their investors, who viewed biotech as a risky bet.

Their attitude didn't start to change until about 2013, by which time the recession was over and advances in drug research had made biotech more attractive to a wider group of investors and potential buyers. Biopharma acquisitions and initial public offerings, typically the two main ways venture firms receive returns, would hit record highs in the following years, giving these firms and their backers the confidence to keep putting in money.

Indeed, since 2013 there's been an annual uptick in the number of funding deals venture firms are doing, with almost every year having about 70 more than the one prior, according to PitchBook. By 2019, the deal count had hit 941.

The collective value of these deals, which range from small angel investments to the larger funding rounds that follow, has grown too. In four of the last five years it surpassed $10 billion.

The favorable conditions also made it so that venture firms could go back to their investors for more money. Polaris Partners, 5AM Ventures, Third Rock Ventures and Versant Ventures, among others, each secured hundreds of millions of dollars across 2018 and 2019, while Flagship, Arch Venture Partners and venBio closed new funds this spring worth almost $3 billion combined.

Deerfield, a type of investor known as a "crossover" because it invests in both private and publicly traded companies, also just completed raising $840 million to put into healthcare companies.

While money has been plentiful, the economic disruption caused by the coronavirus raises doubts about whether that will continue.

Bob Nelsen, managing director at Arch, said he'd be surprised if any new, first-time funds can raise cash at all this year. Firms with existing networks of investor relationships may be able to pull off follow-on funds, he added, but they'd likely take longer to complete.

If a slowdown persists, young biotechs could find it difficult to close their next rounds of financing. Already, the pace of biopharma venture deals appears to be lagging, as PitchBook counted 228 deals between early February and mid-May this year, down from the 271 seen in a similar time frame in 2019.

One top concern is that crossover investors, who often come in later and supply a substantial amount of the funding that props up a company until it goes public, will back away from biotech startups. Without those investors, early-stage venture backers might have to dig deeper in their pockets to push their companies forward.

"It can take $1 billion to get a drug to market," said Kristopher Brown, a partner in the life sciences group at law firm Goodwin. "There are few venture capitalists who can afford to fund that."

Nelsen predicts some crossover investors will take a break from biotech startups and focus on public stocks that are now cheaper because of a turbulent market. But Jon Norris, a managing director at Silicon Valley Bank who works on deals with healthcare venture firms, isn't so sure.

Biotech stocks have held up relatively well this year compared to the rest of the market, which Norris said bodes well for continued crossover interest. What's more, the number of biotechs that have gone public this year 14 as of May 26 is just a tick down from the 17 IPOs completed by the same date in 2019.

"It just means to me that people continue to see this sector as one that's worthy of investing," Norris said. "If you see good returns, people are not going to be quick to exit the market."

After dip, biotech stocks have outperformed the market

XBI vs S&P; 500, values indexed to Jan. 2, 2020=100

Still, much is unknown about how the pandemic will further unfold.

For drug companies, the impact of social distancing and its ripple effects on the economy are expected to be more dramatic in the second and third quarters. In a possibly foreboding sign, industry bellwethers Merck & Co. and Johnson & Johnson have lowered their revenue forecasts for the year by billions of dollars.

"I do worry about the delays that are inherent to having this whole economy come to a stop and hospital systems being overwhelmed," Norris said. "To me, that's a big deal over the next quarter."

In the meantime, venture firms need to put the money they've already raised to work.

Early-stage investors who spoke to BioPharma Dive said their core strategies are still intact in spite of the coronavirus. Flagship and Arch prefer companies with technology platforms that, in theory, can give rise to multiple drugs. Polaris, as it has in the past, works its close relationships with academic institutions to find new startup opportunities. Atlas Venture remains fairly agnostic, while San Francisco-based venBio looks for companies on track to hit a meaningful milestone in the next three to five years.

And yet, the pandemic does weigh on their thinking.

To attract new investors, development partners and potential acquirers, biotech startups need to hit goals like moving a drug into and through human testing. But they've found a new obstacle in the coronavirus. By late May, nearly 100 drug companies of all sizes had reported impacts to their clinical trials related to the pandemic.

"There could be significant dollars lost and significantly extended timelines" for biotechs on the verge of, or already in, clinical testing, said James Flynn, managing partner at Deerfield.

As such, some firms are investing more selectively. Aaron Royston, a managing partner at venBio, said his team will be "very cautious" when putting money into any drug company that's close to starting an important trial or launching a new product.

Funding also might be harder to come by for biotechs built around a single drug program, as there's not much cushioning if that program runs into complications.

"Companies that are purely based on single assets with a clinical readout are in deep shit," Nelsen said.

By contrast, companies at the earliest stages of research may benefit. Investors assume that, by the time these companies reach human trials, some of the challenges and uncertainties surrounding the coronavirus will have been ironed out.

Royston, for instance, said he has little apprehension investing in biotechs that will be working on early research for the next 12 to 18 months.

"Preclinical investment is almost a safe place to hide while everybody else is on the later-stage side, trying to figure out how to deal with delays in clinical trials," SVB's Norris said.

For now, venture firms say they've been more frequently checking in with companies that could face setbacks because of the disruption and, if needed, helping devise plans to conserve cash.

"At the end of the day, data is the currency of how we value our progress," said Atlas Venture partner Bruce Booth. "So, as long as the biotech has enough capital to get it through those data collections and can get out from some of those R&D delays, then I think we'll be in an OK place coming out of this crisis."

In responding to the disruption brought by the pandemic, venture capitalists may revisit approaches honed after the last big economic downturn in 2008.

Then, a dried up IPO market alongside difficulties raising money led some venture firms to leave life sciences investing altogether. Others doubled down on their existing strategies or adopted new ways to build companies.

Versant, for example, was known to start companies with a prearranged buyer in place. Atlas gave some companies, like Nimbus Therapeutics, a limited liability structure that made it easier to sell individual drugs to buyers, though more complicated to go public. Such tools are "less critical now than they were during that challenging period" because biotechs can still conduct IPOs, Booth says.

At Polaris, hard economic times reinforced the firm's trust in a type of group investing called syndicates, which can spread risk between firms. Flagship, on the other hand, backed away from forming biotechs with other investors because the process felt too restrictive.

"What we found was that, when people were traumatized through financing risks and through uncertainty, a syndicate was only as strong as its weakest link," Afeyan said. "In other words, if you had five investors sitting around a board table, the weakest one was the one that got to decide what you did."

Flagship has since shifted resources to focus almost exclusively on creating startups in its own labs. And it isn't alone. Firms such as Third Rock have become known for an intensely hands-on approach, incubating companies and ultimately owning significant stakes when those biotechs go public.

Another popular strategy has been to stagger, or tranche, investments to limit risk. Typically, this means firms give smaller chunks of cash early on and larger chunks later, once a startup has provided more evidence that its medicines might pan out.

And yet, despite the unprecedented challenges posed by the pandemic, venBio and others appear optimistic that a 2008-like shakeout isn't coming, and that they won't have to rely on unorthodox strategies to navigate the future. Royston's view on 2020 opportunities hasn't changed; Nelsen doesn't foresee the pandemic preventing Arch from investing right now; and Flagship is still on track to spin around 10 projects into full companies over the next year and a half, Afeyan said.

There's a key difference this time around, several firms and advisors said, and that's the money which has so far stayed readily available to healthcare investors. Cowen Healthcare Investments just last week finished raising nearly half a billion dollars, adding to the string of recent hauls from other firms.

"We've seen these things come and go, and frankly we've done some of our best companies in the down cycles," Nelsen said.

A pandemic, however, isn't just another down cycle.

Past downturns didn't threaten to overwhelm the healthcare system, as the outbreak of the coronavirus has. Hundreds of thousands of Americans have been sickened by coronavirus infections. And for millions of people with diseases other than COVID-19, how they seek and receive care changed overnight.

The widespread shutdown of businesses across the country, meanwhile, has created economic hardship not seen since the Great Depression, and it's unlikely a stop-and-start reopening will quickly heal those wounds.

"No one fully can comprehend, even in a world as smart as the biotech scientific world, the trajectory and the impact of the current situation," said Amy Schulman, a managing partner at Polaris.

Whether the pandemic persists into next year or lingers much longer, venture capitalists do acknowledge it will have profound effects on society and, by extension, the drug industry.

Nashat envisions that "new kinds of entrepreneurs" will rise amid the chaos, while others will be "scared off." Nelsen predicts big changes in how healthcare is delivered, which will "shock" the system and create new opportunities.

That means investors will need to adapt too.

"It would be incredible, to me," Afeyan said, "if people just forgot this and resumed their old normal."

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Venture capital found its footing in biotech. Then came the virus. - BioPharma Dive

Israeli Lab: Drugs For Gaucher Disease May Work Against Coronavirus, Other Viral Infections | Health News – NoCamels – Israeli Innovation News

Israeli scientists at the Israel Institute for Biological Research (IIBR) have found that a combination of two existing antiviral drugs for Gaucher disease appears to inhibit the growth of SARS CoV-2, the virus that leads to COVID-19 and may work against other viral infections, including a common flu strain.

The IIBR is a governmental research center specializing in biology, chemistry and environmental sciences that falls under the jurisdiction of the Prime Ministers Office. During the pandemic, announcements have been issued by the Defense Ministry.

According to a press announcement on Tuesday, scientists at the secretive bio-defense lab tested an analog of the FDA-approved drug Cerdelga, and an analog of a second drug, Venglustat, currently in advanced trials. They found that, in combination, the drugs led to a significant reduction in the replication capacity of the coronavirus and to the destruction of the infected cells.

The two drugs are used to treat Gaucher disease, an inherited genetic condition most common in people of Ashkenazi Jewish descent that leads to the buildup of fatty substances in certain organs, particularly the spleen and liver, and can affect their function. The disease can also lead to skeletal abnormalities and blood disorders, In rare cases, Gaucher disease can also lead to brain inflammation, according to the Mayo Clinic. The disease is unrelated to COVID-19.

The Israeli researchers tested the drugs on mouse models using four different RNA viruses: Neuroinvasive Sindbis virus (SVNI), an infection transmitted via mosquitos that can lead to years of debilitating musculoskeletal symptoms; West Nile virus (WNV), also a mosquito-borne disease that can cause neurological disease and is potentially fatal; Influenza A virus, a strain of the flu; and SARS-CoV-2.

The researchers found that the two drugs were effective in all four cases. They work by inhibiting glucosylceramide synthase *GCS), an enzyme involved in the production of glucocerebroside, a lipid that accumulates in the tissues of patients affected with Gaucher disease. In the lab setting, they inhibited the replication of the viruses, and in the case of mice infected with SVNI, increased their survival rate.

In the case of COVID-19, the drugs have an antiviral effect on the SARS-CoV-2 clinical isolate in vitro, with a single dose able to significantly inhibit viral replication within 2448 h.

The two drugs are currently being tested for their effectiveness in treating animals infected with the coronavirus.

The study, published in bioRxiv, has not yet been peer-reviewed. The authors are all from the IIBRs Department of Infectious Diseases

The data suggests that GCS inhibitors can potentially serve as a broad-spectrum antiviral therapy and should be further examined in preclinical and clinical trial, the scientists wrote, adding that repurposing approved drugs can lead to significantly reduced timelines and required investment in making treatment available.

Treatment of a new disease such as COVID-19 using an existing, approved drug may serve as an effective short-term solution considering that one of the major challenges in addressing such a pandemic is the length of time it takes for both the research and approval phases of new drugs, the Defense Ministry wrote in the announcement.

The lab has been conducting various research into COVID-19 for several months, including studies on possible treatment and a vaccine. Israeli Prime Minister Benjamin Netanyahu tapped the institute in early February to begin development on inoculation. In early April, the center reported significant progressand trials on animals.

The institute has also been involved in plasma collection from Israelis who have recovered from COVID-19 to research antibodies, proteins made by the immune system that can attack the virus.

Earlier this month, the IIBR said it completed a groundbreaking scientific development toward a potential treatment based on an antibody that neutralizes SARS-CoV2. The development had three key parameters, according to the IIBR: first, the antibody is monoclonal (lab-made identical immune cells that are all clones of a unique parent cell), and contains a low proportion of harmful proteins; second, the institute has demonstrated the ability of the antibody to neutralize the coronavirus; and third, the antibody was specifically tested on SARS CoV-2.

The Ness Ziona-based institute said it is now pursuing a patent for its development after which it will approach international manufacturers.

A number of Israeli scientific teams and over 100 groups worldwide are currently working to develop a vaccine or a treatment for COVID-19.

At least 10 candidate vaccines are in clinical evaluation, including those of Massachusetts-based company Moderna which was the first to develop an experimental vaccine that went into trial quickly, and California-based biotech firm Gilead Sciences, which is evaluating the safety and efficacy of its novel antiviral drug Remdesivir, developed originally for Ebola, in adults diagnosed with COVID-19.

Last month, Israeli scientists at theMigal Galilee Research Institute formed a new company, MigVax, to further adapt a vaccine they developed for a deadly coronavirus affecting poultry for human use. The scientists had been working for four years to develop a vaccine for IBV (Infectious Bronchitis Virus) which affects the respiratory tract, gut, kidney and reproductive systems of domestic fowl.

Also in April, an Israeli scientist wasawarded a US patent for his innovative vaccine design for the corona family of viruses and indicated that he was on track to develop a vaccine for SARS CoV2.

Meanwhile, two Israeli bio-medical companies nabbed FDA approval for separate trials in the US with their respective solutions for COVID-19 as part of a compassionate use program, a treatment option that allows for the use of not-yet-authorized medicine for severely ill patients.

BothRedHill BioPharma, a publicly-traded specialty biopharmaceutical company, andPluristem Therapeutics, also a public company that specializes in placental cell therapy, were given the green light for their imminent separate studies with the investigational drug, opaganib, and the placental cell therapy PLX, respectively.

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Israeli Lab: Drugs For Gaucher Disease May Work Against Coronavirus, Other Viral Infections | Health News - NoCamels - Israeli Innovation News

Rapid Growth on Genetic Testing Market with COVID-19 Impact Analysis, Top Key Companies like Abbott Laboratories Bio-Rad Laboratories Inc.,…

The Global Genetic Testing Market is expected to register substantial growth in the near future due to rise in incidence of genetic disorders & cancer and growth in awareness and acceptance of personalized medicines.

Genetic testing is also known as DNA testing. Genetic testing is the study of gene present in cells and tissues. This study is further applied in the field of biology and medicine to better understand genetic disorders such as cancer, sickle cell anemia, cystic fibrosis, Down syndrome, and others. The scope of the report discusses the use of gene tests for the development of personalized medicine, targeted cancer treatment, and other genetic diseases.

In addition, advancements in genetic testing techniques and increasing application of genetic testing in oncology are expected to boost the market growth during the forecast period.Genetic tests involve a set of lab tests for the study of the genetic makeup of patients and identify any gene mutations and alterations in the healthy structure of DNA leading to the development of genetic disorders. The Geographical Segmentation includes study of global regions such as North America, Latin America, Asia-Pacific, Africa, and Europe. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study.

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Genetic Testing Key Market Segments:

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Different top-level key players are also enlisted in order to obtain in-depth knowledge and informative data of companies. Some of the key players are also profiled in this research report, which includes Genetic Testing Market. Different industry analysis tools such as SWOT and Porters five-technique are further used while analyzing the global Genetic Testing Market.

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The report also draws attention to recent advancements in technologies and certain methodologies which further help to boost the outcome of the businesses. Furthermore, it also offers a comprehensive data of cost structure such as the cost of manpower, tools, technologies, and cost of raw material. The report is an expansive source of analytical information of different business verticals such as type, size, applications, and end-users.

Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market.

Table of Content:

Chapter 1:Genetic Testing Market Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3:Genetic Testing Market Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Genetic Testing Market Effect Factors Analysis

Chapter 12: GlobalGenetic Testing Market Forecast to 2025

Finally, all aspects of the Genetic Testing Market are quantitatively as well qualitatively assessed to study the Global as well as regional market comparatively. This market study presents critical information and factual data about the market providing an overall statistical study of this market on the basis of market drivers, limitations and its future prospects.

About Us:Market Research Inc is farsighted in its view and covers massive ground in global research. Local or global, we keep a close check on both markets. Trends and concurrent assessments sometimes overlap and influence the other. When we say market intelligence, we mean a deep and well-informed insight into your products, market, marketing, competitors, and customers. Market research companies are leading the way in nurturing global thought leadership. We help your product/service become the best they can with our informed approach.

Contact Us:Author KevinUS Address: 51 Yerba Buena Lane, Ground Suite,Inner Sunset San Francisco, CA 94103, USACall Us: +1 (628) 225 1818Email: [emailprotected]

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Rapid Growth on Genetic Testing Market with COVID-19 Impact Analysis, Top Key Companies like Abbott Laboratories Bio-Rad Laboratories Inc.,...

insideHPC Special Report: HPC and AI for the Era of Genomics – insideHPC

This special report sponsored by Dell Technologies, takes a deep dive into HPC and AI for life sciences in the era of genomics. 2020 will be remembered for the outbreak of the Novel Coronavirus or COVID-19. While infection rates are growing exponentially, the race is on to find a treatment, vaccine, or cure. Governments and private organizations are teaming together to understand the basic biology of the virus, its genetic code, to find what can stop it.

Significant amounts of computing power are aimed at this problem, including using the most powerful high performance computing (HPC) systems in the world today. Finding a cure or eliminating COVID-19 will not only benefit the worldwide population, but will also be the foundation for tackling the next pandemic, which some scientists say will happen in the not too distant future.

This technology guide, insideHPC Special Report: HPC and AI for the Era of Genomics, highlights a lineup of Ready Solutions created by Dell Technologies which are highly optimized and tuned hardware and software stacks for a variety of industries. The Ready Solutions for HPC Life Sciences have been designed to speed time to production, improve performance with purpose-built solutions, and scale easier with modular building blocks for capacity and performance.


2020 will be remembered for the outbreak of the Novel Coronavirus or COVID-19. While infection rates are growing exponentially, the race is on to find a treatment, vaccine, or cure. Governments and private organizations are teaming together to understand the basic biology of the virus, its genetic code, to find what can stop it. Significant amounts of computing power are aimed at this problem, including using the most powerful high performance computing (HPC) systems in the world today.[1] Finding a cure or eliminating COVID-19 will not only benefit the worldwide population, but will also be the foundation for tackling the next pandemic, which some scientists say will happen in the not too distant future.[2]

Artificial Intelligence (AI)

Slav Petrovski, Head of Genome Analytics and Informatics at AstraZenecas Centre for Genomics Research (CGR) explains that there is a wide range of uses for AI within this field. He says that the approximately three billion base pairs that make up the human genome can be analyzed through AI to find genetic variations. The next step is to determine the level of confidence to be placed in the differing data to decide if it represents a biological genetic variant.

AI is being combined with traditional HPC simulations to predict more accurate results. Based on previous completed computations, an algorithm is able to determine what the next input or result could be. For example, if protein A shows a possible affinity to kill the Coronavirus, then does protein B (or others) have a better chance or could be more effective?

There are a number AI uses within genomics research that can identify and facilitate drug target interaction. By combining analytical and automated processes with the ongoing study of genomics, a more complete understanding of this field will progress. AI can lead to greater insights into the patterns and anomalies in the data, where humans may not see the correlations at first. By using machine and deep learning techniques, new and more effective medicines can get to patients faster and will be better targeted to fight diseases.

Produvia, a research company, has identified five areas where AI will benefit genomic research moving forward:

Precision Medicine

While AI can benefit genomic research, the end goal is to create treatments that specifically attack the genetic code of the infection or disease, and to create treatments that are tailored to an individuals genetic makeup. To do this requires significant data, computing power, and collaborations that combine expertise from many disciplines.

With a combination of faster and more accurate genomic sequencing with faster computer systems and new algorithms, the movement of discovering what medicine will work best on individual pathogens and patients has moved from research institutions to bedside doctors. Physicians and other healthcare providers now have better, faster and more accurate tools and data to determine optimal treatment plans based on more data. This is especially true for pediatric cancer patients.

Personalized or precision medicine holds the key to innovative approaches to manage diseases on an individual level. Various decisions regarding the management of healthcare to each pathogen and/or individual is customized, based on the knowledge of the genetic or cellular information. Diagnosis of diseases, and the resulting treatments can be tailored for each person. However, a number of challenges exist as this scientific field moves forward, such as regulatory oversight, intellectual property rights and patient privacy.

Worldwide, many countries are dedicating resources and efforts to learn more about genomics and how to apply this knowledge to personalizing medicine. Figure 1 shows the worldwide effort to bring precision medicine to those in need. Figure 2 shows how the cost of decoding a human genome has come down, even faster than Moores Law.

Over the next few weeks we will explore these topics surrounding HPC and AI for life sciences in the era of genomics:

Download the complete insideHPC Special Report: HPC and AI for the Era of Genomics, courtesy of Dell Technologies.

[1] COVID-19 HPC Consortium

[2] Scientists in race to protect humanity from future pandemics

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insideHPC Special Report: HPC and AI for the Era of Genomics - insideHPC

More insight into the cytokine storm caused by Covid-19 could lead to a treatment – Health24

The immune system response triggered by Covid-19, causing an overproduction of cytokines, has been big news during the pandemic.

While Covid-19 deaths are usually caused by acute respiratory distress syndrome (ARDS), especially in older adults and those with co-morbidities, some younger Covid-19 patients have suffered severe symptoms because of an overreaction by their immune systems, rather than the virus itself.

Now, a new clinical trial will test a treatment that targets this immune response, according to a press release from the Howard Hughes Medical Institute.

The mechanism behind the cytokine storm

According to leading immunologists in Japan, a molecular mechanism could lead to possible ways to treat this overreaction by the immune system. The research was published in the journal Immunity.

The immune system response to the coronavirus may lead to ARDS, causing patients to struggle for oxygen in their inflamed, fluid-filled lungs.

"To rescue the patients from this condition, it is vital to understand how SARS-CoV-2 triggers the cytokine storm that leads to ARDS," stated Masaaki Murakami, the head of the immunology laboratory at Hokkaido University's Institute for Genetic Medicine.

His study suggested that the novel coronavirusenters human cells by attaching to the ACE2 surface receptor. Then, a human enzyme called TMPRSS2 is utilised.

"Drugs that block the ACE2 receptor or that inhibit the enzyme could help treat the initial stages of the disease," says Murakami. "However, ARDS with cytokine storm starts to appear in the later phase of infection even when the numbers of the virus decrease. So, there must be another pathway that causes the cytokine storm, Murakami explained in a news statement.

Closer to a treatment?

The treatment that will be tested in a clinical trial by the Howard Hughes Medical Institute involves a common type of alpha-blocker. Through mouse studies, the team determined that this drug might break the hyperinflammation before it causes the severe symptoms seen in Covid-19 patients.

"The approach we're advocating involves treating people who are at high risk early in the course of the disease, when you know they're infected but before they have severe symptoms. If the trial's results suggest the drug is safe and effective against Covid-19, it could potentially help many people recover safely at home and lessen the strain on hospital resources, stated Howard Hughes Medical Investigator Bert Vogelstein.

Together with his team at the John Hopkins University School of Medicine, Vogelstein is currently recruiting patients aged 45 to 85 at the John Hopkins Hospital to participate in the trial. The prerequisites are that they have to be hospitalised, but not ventilated or in ICU.

How will an alpha blocker stop the cytokine storm?

A hyperactive immune system isnt a new response solely seen in Covid-19. Usually, this type of response is seen in people already suffering from autoimmune diseases or cancer.

What happens during a cytokine storm is that cells called macrophages, which are either found in the tissues or in the blood as white blood cells, are activated to detect and fight the pathogen. As soon as this happens, cytokines are released to help the body fight off the intruder.

Unfortunately, the macrophages dont only release cytokines, but also molecules called catecholamines, which trigger the immune system to release even more cytokines.

According to the news release, Vogelsteins team was already investigating how this reaction in cancer patients could be halted with immunotherapy.

They then looked at alpha-blockers which are usually prescribed for prostate conditions and high blood pressure. This medication is meant to help curb the cells that trigger cytokine storms.

The initial research in mice was published in the journal Nature in 2018.

How likely is this method to be successful?

While alpha-blockers were already approved for human use, Vogelsteins team needed to look at medical claims data to see how patients with pneumonia and ARDS responded to alpha-blockers for unrelated conditions.

The conclusion was that the use of alpha-blockers were correlated to lower death risk, but this simply wasnt enough evidence for a new condition such as Covid-19.

Now, the patients on trial will take increasing doses of an alpha-blocker over six days. The team will then evaluate whether those patients had lower risk of ICU admission and being placed on ventilators.

A second trial will be needed to establish whether this approach is safe and effective. According to Vogelstein, this method may be great for helping to mitigate symptoms before they become severe and deadly.

"Eventually, hopefully, a vaccine will be produced, and that will be the essence of prevention," he stated. "But until vaccines are available, secondary prevention makes a lot of sense."

Image credit: Getty Images

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More insight into the cytokine storm caused by Covid-19 could lead to a treatment - Health24

4000 Years of contact, conflict and cultural change had little genetic impact in Near East – University of Birmingham

A 2,000-year-old individual sequenced in the study. Photo credits: Directorate General of Antiquities (Lebanon)

The Near East was a crossroad for the ancient worlds greatest civilizations, and invasions over centuries caused enormous changes in cultures, religions and languages. However, a new study of the DNA of ancient skeletons spanning 4,000 years has revealed that most of these changes had no lasting effect on the genetics of the local population of Beirut.

Whilst the invasions and conquests may have been revolutionary for the elite rulers, researchers at the Wellcome Sanger Institute, University of Birmingham, French Institute of the Near East in Lebanon and their collaborators found only three time periods that had any impact on the long-term genetics of the ordinary people. These were the beginning of the Iron Age, the arrival of Alexander the Great, and the domination of the Ottoman Empire.

Reported today (28 May) in the American Journal of Human Genetics, the study shows the value of using genetics alongside archaeology to help understand what could be happening in the lives of ordinary people throughout history.

Over the centuries, the Levant has had many different rulers, including the Egyptians, Babylonians, Assyrians, Persians, Greeks, Romans, Crusaders, Arabs, and Ottomans. Most of these had permanent cultural effects on the local population, including changes to religion and even languages, as shown by the historical records and archaeological findings.

However, despite this, previous research showed that present-day local people in Lebanon were mainly descended from local people in the Bronze Age (2100-1500 BCE), with 90 per cent of their genetic make-up coming from around 4,000 years ago, and very few lasting traces of even the Crusaders invasion around the 11th-13th Century.

To understand this potential contradiction and build a picture of the genetic history of ordinary people in the region, the researchers studied the DNA of ancient skeletons through 4,000 years. The team sequenced the genomes of 19 ancient people who lived in Lebanon between 800BCE and 200CE, and by combining with previous ancient and modern data, created an 8-point time line across the millennia.

Scientists detected lasting genetic changes in the local people from just three time periods - during the beginning of the Iron Age (about 1,000 BCE), the arrival of Alexander the Great (beginning 330 BCE), and the domination of the Ottoman Empire (1516 CE) but not from the other times.

Dr Marc Haber, first author from the University of Birmingham and previously from the Wellcome Sanger Institute, said: We revealed a genetic history of the area across 4,000 years, with a time-point approximately every 500 years. This showed us that despite the huge cultural changes that were occurring during this period, there were only a few times that the genetics of the general population changed enough to affect the ordinary people.

The study revealed that some people did mix and form families with people from other cultures. One burial site was found to contain the remains of an Egyptian mother, and her son whose father had Egyptian and Lebanese ancestry. However, this cosmopolitan mixing did not seem to be widespread.

Historical evidence is based on archaeological findings and written records, but these are biased towards the elite rulers and people with money and influence, as they have far more resources and write the history. It can be difficult to understand the lives of the ordinary people.

Dr Joyce Nassar, an author on the paper and archaeologist from the French Institute of the Near East, Lebanon, said: This study is really exciting, as the genetic evidence is helping us to interpret what we find. Some people might think that when a land was invaded, that the population would change. But this study shows it isnt that simple, and reveals there was only limited biological mixing, despite the cultural and political influence of the invasions.

The skeletons came from four archaeological excavation sites in Beirut, which were discovered during building projects in the Lebanese capital city and rescued by the Directorate General of Antiquities*. The archaeologists and researchers then worked together to transfer the bones to a laboratory in Estonia dedicated to ancient DNA, where the surviving ancient DNA was extracted from the temporal bone in the skulls. The DNA was then sequenced and analysed at the Sanger Institute. Recent advances in DNA extraction and sequencing technology made studying the ancient and damaged DNA possible.

Dr Chris Tyler Smith, senior author on the paper and previously from the Wellcome Sanger Institute, said: We see that people like the Egyptians and the Crusaders came to Lebanon, lived, raised families and died there. Their DNA sequences reveal this, but a little while later, there may be no trace of their genetics in the local population. Our study shows the power of ancient DNA to give new information about the human past, that complements the available historical records, and reveals the benefits of archaeologists and geneticists working together to understand historical events.

For more information or interviews, please contact:Hasan Salim Patel, Communications Manager (Arts, Law and Social Sciences) or contact the press office out of hours on +44 (0) 7789 921 165.

Or Contact:Dr Samantha Wynne, Media OfficerWellcome Sanger Institute,Phone: +44 (0)1223 492368

*Directorate General of Antiquities (DGA) at the Ministry of Culture is the authority responsible for the Lebanese heritage, including all the excavations led on the Lebanese territories, museums and historical buildings and cities. The DGA gave the permission to conduct DNA analysis on the material and to publish it.

About the University of Birmingham

The University of Birmingham is ranked amongst the worlds top 100 institutions. Its work brings people from across the world to Birmingham, including researchers, teachers and more than 6,500 international students from over 150 countries.

Publication:Marc Haber et al. (2020) A genetic history of the Near East from an aDNA time course sampling eight points in the past four thousand years. American Journal of Human Genetics. DOI: 10.1016/j.ajhg.2020.05.008

Funding:This work was supported by Wellcome (098051); the European Union through the European Regional Development Fund [Project No. 20142020.4.01.160030]; and the Estonian Research Council [PRG243].

The French Institute of Near East, Lebanon / Institut Francais du Proche-Orient (IFPO)The French Institute of Near East, a research centre of international renown, is organized into 3 scientific departments: Archaeology and History of Antiquity (DAHA), Arabic, Medieval and Modern Studies (DAMM) and Contemporary Studies (DC). From Beirut, where its headquarters are located, IFPO has regional competence over 5 countries with branches in Jordan (Amman), the Palestinian territories, Iraq (Erbil), and Syria (Damascus and Aleppo). The research carried out at IFPO concerns many disciplines in Human and Social Sciences, carried out through a multidisciplinary and cross-period approach aiming to understand the societies of the Near East from prehistory to the present days. For more information, visit the websiteor follow us on Twitter, Facebook, LinkedIn, and Instagram.

The Wellcome Sanger Institute

The Wellcome Sanger Institute is a world leading genomics research centre. We undertake large-scale research that forms the foundations of knowledge in biology and medicine. We are open and collaborative; our data, results, tools and technologies are shared across the globe to advance science. Our ambition is vast we take on projects that are not possible anywhere else. We use the power of genome sequencing to understand and harness the information in DNA. Funded by Wellcome, we have the freedom and support to push the boundaries of genomics. Our findings are used to improve health and to understand life on Earth. Find out more at http://www.sanger.ac.uk or follow us on Twitter, Facebook, LinkedIn and on our Blog.

About Wellcome

Wellcome exists to improve health by helping great ideas to thrive. We support researchers, we take on big health challenges, we campaign for better science, and we help everyone get involved with science and health research. We are a politically and financially independent foundation.

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4000 Years of contact, conflict and cultural change had little genetic impact in Near East - University of Birmingham

UCalgary researchers launch 360-degree study of children and COVID-19 – UCalgary News

When it comes to children and COVID-19, we have many more questions than answers. Why are most children not getting as sick from the virus? Why are some becoming critically ill? Why are some developing puzzling symptoms? And, are children carrying the virus and spreading it silently through families and communities?

A multidisciplinary team of UCalgary researchers is studying the genes and immune response of Alberta children, and the unique genetic code of the virus itself in pan-Alberta research focused on tracking the transmission of COVID-19.

Children here in Alberta, and around the world, have milder symptoms and recover more rapidly than adults with COVID, says Dr. Jim Kellner, MD(pictured above), a professor in the departments of Paediatrics, Microbiology, Immunology and Infectious Disease, and Community Health Sciences at the Cumming School of Medicine (CSM), and an infectious disease researcher at the CSMs Alberta Childrens Hospital Research Institute (ACHRI).

We want to better understand how contagious children are, precisely how the virus is affecting their young bodies, and how children develop immunity against COVID-19.

Kellner is leading a team of 29 child health and wellness scientists and physicians to study children with COVID-19 across the province. The Alberta Childhood COVID-19 Cohort Study (AB3C) will investigate how children are responding to the infection and how they are spreading it. The UCalgary study, a collaboration between the University of Alberta, Alberta Health Services (AHS), the Alberta Childrens Hospital and Alberta Precision Laboratories (APL), is funded by Genome Alberta and the Alberta Childrens Hospital Foundation through ACHRI.

Alberta is a Canadian leader in COVID testing, completing more than 200,000 tests, testing more than 22,000 children and youth, including some with no symptoms, says Kellner, also a member of the Calvin, Phoebe and Joan Snyder Institute for Chronic Diseases, and the OBrien Institute for Public Health.

We are so fortunate in Alberta to have province-wide systems and teams AHS, APL, and electronic medical records that facilitate the study of the virus infection in children and detection of antibodies in children who have contracted the disease.

This research will draw from provincial COVID test results and collect blood, urine and stool samples which will be biobanked. Kellners team will then follow up with families to track short- and long-term symptoms of the disease and its impact on family and community members.

A member of the Canadian COVID-19 Immunity Task Force Leadership Group, Kellner will report Alberta findings to a national network of scientists, clinicians and public health experts. This powerful collaboration between a national initiative and UCalgary scientists will allow us to share detailed evidence on COVID-19 spread, the development of immunity and targets for potential treatments and vaccines, says Kellner.

Francois Bernier, left, with David Bailey of Alberta Genome.

Riley Brandt, University of Calgary

The team will also look at the biology of the virus, the childs genome and immune response to the infection. One aim of this deep dive is to study whether the virus is changing as it moves through the population. Dr. Francois Bernier, MD, head of the Department of Medical Genetics and professor in the Department of Paediatrics at the CSM and an ACHRI physician-scientist, is leading the genomics arm of the study.

Bernier and his colleagues will delve into the unique bar code of each instance of the virus itself. The genetic code of the virus is like a FedEx label, says Bernier. Deep analysis of the viruss genes will allow us to precisely trace transmission, allowing us to say This one originated in Europe, and this one came from the United States, he says.

Investigating COVID-19 in children will help scientists understand why some people become critically ill with the virus and others only mildly ill. To that end, the genomics team will also investigate the full biology of Albertans infected with the virus. The interplay between the viruss genes and the genes of infected Albertans is a key focus.

Not everyone is equally susceptible to COVID, says Bernier. We suspect genetics play a critical role.

Data mining is also essential to our understanding the complexities of COVID, he continues. We have big data experts at UCalgary who will use machine learning to give us real answers on this virus, how it is moving and spreading, and how it is interacting with the genes and the immune system of Albertans who are infected.

The multi-disciplinary team includes immunologists, epidemiologists, infectious and inflammatory disease experts, geneticists, bioinformaticians, health economists, and advanced computing experts. Bernier will share his teams data with national and international research groups including GISAID, contributing to a global effort to map the pandemics movement, its evolving genetics, and the latest development of medical countermeasures.

As social isolation policies change and we focus more keenly on community spread, it will be increasingly important to identify, quantify and track infections, immunity, and disease severity to be better able to predict and prevent the risk to children and families, says Kellner.

Information on UCalgarys response to COVID-19 can be found on the Emergency Management website.

The University of Calgary is uniquely positioned to find solutions to key global challenges. Through the research strategy forInfections, Inflammation, and Chronic Diseases in the Changing Environment (IICD), top scientists lead multidisciplinary teams to understand and prevent the complex factors that threaten our health and economies.

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UCalgary researchers launch 360-degree study of children and COVID-19 - UCalgary News

Precision Medicine Market Overview By Growing Demands, Trends And Business Opportunities 2020 To 2027 – Cole of Duty

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Precision Medicine market.

Trusted Business Insights presents an updated and Latest Study on Precision Medicine Market 2019-2026. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Precision Medicine market during the forecast period (2019-2029).It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Precision Medicine Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19)

Abstract, Snapshot, Market Analysis & Market Definition: Precision Medicine MarketIndustry / Sector Trends

Precision Medicine Market size was valued at USD 52.6 billion in 2018 and is expected to witness 10.5% CAGR from 2019 to 2025.

U.S.Market Segmentation, Outlook & Regional Insights: Precision Medicine Market

Precision Medicine Market, By Technology, 2018 & 2025 (USD Million)

Growing demand and advancements in cancer biology will drive personalized medicine market during the forecast period. Accessibility to large-scale human genome database including next-generation sequencing (NGS) and computational tools foster industry growth. Development of innovative genetic technologies examines the functional effect of genetic makeup that leads in developing cancer, thus, should propel huge demand for cancer biology. Several such tools are widely used to study the mechanism of DNA repair, epigenetic changes related to cancer and gene regulation in cancerous cells that offer opportunities for cancer biology in personalized medicine.

Moreover, improving efficiencies within the health care system will serve to be positive impact rendering factor personalized treatment business growth. Benefits offered by personalized medicines and treatment includes target treatment for patient, optimal dosing, focus on prevention and earlier intervention as well as preventing adverse events. Thus, above mentioned features will foster the demand for personalized medication market growth. However, high cost associated with precision medicine may impede industry growth over the forecast period.

Precision Medicine Market, By Technology

Drug discovery segment held over 21% revenue share in 2018 and is projected to grow significantly by 2025. Focus of business players on developing technologically advanced drugs enabling superior treatment for several life-threatening diseases will create segmental growth opportunities. Bio-pharmaceutical companies utilize bioinformatics software to introduce customized novel drugs that should augment the segmental growth.

Gene sequencing segment is anticipated to show exponential CAGR of around 11% over the coming years. Benefits offered by gene sequencing technique such as relevant information about patients genome and biological research ensures quick drug discovery process that should boost segmental growth.

Precision Medicine Market, By Application

Immunology segment accounted for over USD 9.5 billion in 2018 and is estimated to witness considerable growth trend during the analysis period. Growing demand for bioinformatics and big data analytics to segregate human genome data obtained from immunological processes favors segmental growth.

Oncology segment held significant revenue share in 2018 and is assessed to show more than 10.5% CAGR during the forecast period. Segment growth is attributable to increasing prevalence of cancer cases resulting in development of innovative drugs with specific drug formulations in precision medicine.

Germany Precision Medicine Market Size, By Application, 2018 (USD Million)

Precision Medicine Market, By End-use

Healthcare IT companies segment held more than 27% revenue share in 2018 and will show lucrative growth over coming years. Increasing demand for developing technologically advanced tools for rapid integration, storage, and analysis of patient data should augment the business growth.

Diagnostic companies segment was valued around USD 9.5 billion in 2018. Such companies provide solutions that bridges the gap between clinical needs of patients and technology. Various benefits such as high quality of the medical diagnostic devices enabling precise patient management will foster industry growth.

Precision Medicine Market, By Region

North America precision medicine market is predictable to show around 9% CAGR over the analysis period. Rising prevalence of respiratory and oncology diseases as a result of lifestyle changes is the key factor driving demand for market. Rising healthcare expenditure coupled with presence of major industry players in North America will spur the business growth.

Asia Pacific precision medicine industry was valued more than USD 11 billion revenue in 2018. Regional growth can be attributed to technological advances in sequencing technology. Moreover, presence of large patient pool in Asia Pacific along with growing investments in R&D activities will accelerate personalized medication business growth.

MEA Precision Medicine Market Size, By Country, 2025 (USD Million)

Key Players, Recent Developments & Sector Viewpoints: Precision Medicine Market

Few of the prominent industry players operating in precision medicine industry include Biocrates Life Sciences, Tepnel Pharma Services, Qiagen, Menarini Silicon Biosystems, Novartis, NanoString Technologies, Pfizer, Eagle Genomics, Quest Diagnostics, Roche, Intomics, and Teva Pharmaceutical. The business players implement several strategies including acquisitions, partnerships and innovative product enhancement to capitalize on market growth opportunities.

Recent industry developments:

In October 2018, Eagle Genomics partnered with Microsoft Genomics to tackle computational challenges of genomics era. This partnership aimed to inculcate scale and power of cloud to precision medicine, across the production of fundamental research and core services.

In January 2018, Syapse collaborated with Roche in order to advance precision medicine in oncology. Also, this partnership focuses on clinical delivery and product development for introducing precision medicine to more patients.

Precision Medicine Industry Viewpoint

History of precision medicine can be tracked back in 1950s when Watson and Crick discovered the structure of the DNA as double-helix. Efforts to supplement the DNA structure, researchers cracked the genetic code in early 1960s. Additionally, introduction of the first DNA sequencing technology was developed in 1970s where researchers discovered first enzyme linked to individual variation in response to dosing. In early 1980s Polymerase Chain Reaction (PCR) was first discovered allowing for fast amplification of DNA sequences. These advances continued in 1990s where human genome project was launched along with FDA approval for first personalized medicine with a companion diagnostic, for the treatment of HER2 positive breast cancer. Such form of advancements continued in 2000s where first targeted therapies for lung cancer, leukemia, melanoma, cystic fibrosis, HIV, and other diseases accelerated tremendous growth opportunities for precision medicine. Moreover, increasing demand for personalized medication for secured patient management will drive industry growth during the forthcoming years

Key Insights Covered: Exhaustive Precision Medicine Market1. Market size (sales, revenue and growth rate) of Precision Medicine industry.2. Global major manufacturers operating situation (sales, revenue, growth rate and gross margin) of Precision Medicine industry.3. SWOT analysis, New Project Investment Feasibility Analysis, Upstream raw materials and manufacturing equipment & Industry chain analysis of Precision Medicine industry.4. Market size (sales, revenue) forecast by regions and countries from 2019 to 2025 of Precision Medicine industry.

Research Methodology: Precision Medicine Market

Quick Read Table of Contents of this Report @ Precision Medicine Market Research Report Forecast to 2029 (Includes Business Impact of COVID-19)

Trusted Business InsightsShelly ArnoldMedia & Marketing ExecutiveEmail Me For Any ClarificationsConnect on LinkedInClick to follow Trusted Business Insights LinkedIn for Market Data and Updates.US: +1 646 568 9797UK: +44 330 808 0580

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Precision Medicine Market Overview By Growing Demands, Trends And Business Opportunities 2020 To 2027 - Cole of Duty

Precision Medicine Informs Cost-Effective Heart Disease Treatments – HealthITAnalytics.com

May 18, 2020 -Using precision medicine approaches to tailor heart disease therapies could lead to more cost-effective treatments and improved patient outcomes, according to a study led by researchers at the University of Alabama at Birmingham (UAB).

Patients who experience a heart attack have sharply reduced blood flow in coronary arteries, as well as a high risk of heart failure or death. Coronary angioplasty, a procedure to open narrowed or blocked arteries in the heart, and percutaneous coronary intervention (PCI) can restore blood flow to minimize heart damage. These procedures reduce the risk of subsequent major adverse cardiovascular events (MACE), which include heart attacks, strokes, or death.

After these procedures, providers have to make a treatment decision. After PCI, all patients receive two antiplatelet agents for up to one year. The most commonly used antiplatelet combination after PCI is aspirin and clopidogrel. Clopidogrel is converted to its active form by an enzyme called CYP2C19, but patients respond to this treatment differently depending on their genetic makeup.

Over 30 percent of people have loss-of-function variants in the CYP2C19 gene that decreases the effectiveness of clopidogrel. These patients may not get the full benefit of clopidogrel, which would increase their risk of MACE. The FDA recommends that providers consider different treatments for these individuals, such as prasugrel or ticagrelor, to replace clopidogrel.

In 2018, UAB and researchers at nine universities across the US showed that patients with loss-of-function variants who were treated with clopidogrel had elevated risks. The study revealed that there was a twofold risk of MACE in PCI patients, and a threefold risk for MACE among patients with acute coronary syndrome who received PCI, as compared to patients prescribed prasugrel or ticagrelor instead of clopidogrel.

While prasugrel and ticagrelor are not influenced by loss-of-function variants and can substitute for clopidogrel, these drugs are much more costly and can bring a higher risk of bleeding.

Using this real-world data, the research team set out to conduct an economic analysis of the best treatments for heart disease patients. The study compared three main strategies: treating all patients with clopidogrel, treating all patients with ticagrelor, and genotyping all patients and using ticagrelor in those with loss-of-function variants.

The group considered differences in event rates for heart attacks and stent thrombosis in patients receiving clopidogrel versus ticagrelor versus genotype-guided therapy, during the one-year period following PCI. They also considered medical costs from events like admissions, procedures, medications, clinical visits, and genetic testing. The study used an economic measure known as the quality-adjusted life year (QALY).

First, we looked at which strategy provided the highest QALY, Limdi said. The QALY is the gold standard for measuring benefit of an intervention in our case, genotype-guided treatment compared to treatment without genotyping. Universal ticagrelor and genotype-guided antiplatelet therapy had higher QALYs than universal clopidogrel so those are the best for the patient.

Researchers then analyzed whether those interventions that have higher QALYs were also reasonable from a cost perspective, which includes a payers or patients willingness to pay.

In our case, the payor would recognize that ticagrelor is more expensive than clopidogrel $360 per month vs. $10 per month and there is a $100 cost for each genetic test, Limdi said. So, from the payor perspective, the more effective strategy (one with a higher QALY) if more expensive (higher cost) would have to lower the risks of bad outcomes like heart attacks and strokes for the gains in QALY that are at, or below, the willingness-to-pay threshold.

A measure called incremental cost-effectiveness ratios (ICERs) assesses the incremental cost of the benefit, or improvement in QALY. In the US, a treatment is considered cost-effective if its associated ICER is at or below the willingness-to-pay threshold of $100,000 per QALY.

In our assessment, the two strategies with the highest QALY had very different ICERs, Limdi said. The genotype-guided strategy was cost-effective at $42,365 per QALY. Universal ticagrelor was not; it had an ICER of $227,044 per QALY.

The study results demonstrate the effectiveness of genotyping and precision medicine strategies for tailoring treatments and improving patient outcomes.

We showed that tailoring antiplatelet selection based on genotype is a cost-effective strategy, said Nita Limdi, PharmD, PhD. Support is now growing to change the clinical guidelines, which currently do not recommend genotyping in all cases. Evidence like this is needed to advance the field of precision medicine.


Precision Medicine Informs Cost-Effective Heart Disease Treatments - HealthITAnalytics.com

Colonizing Mars may require humanity to tweak its DNA – Space.com

If humanity is ever going to settle down on Mars, we may need to become a little less human.

Crewed missions to Mars, which NASA wants to start flying in the 2030s, will be tough on astronauts, exposing them to high radiation loads, bone-wasting microgravity and other hazards for several years at a time. But these pioneers should still be able to make it back to Earth in relatively good nick, agency officials have said.

It might be a different story for those who choose not to come home, however. If we want to stay safe and healthy while living permanently on Mars, or any other world beyond our home planet, we may need to make some tweaks to our species' basic blueprint, experts say.

Related: Space radiation threat to astronauts explained (infographic)

Genetic engineering and other advanced technologies "may need to come into play if people want to live and work and thrive, and establish their family, and stay on Mars," Kennda Lynch, an astrobiologist and geomicrobiologist at the Lunar and Planetary Institute in Houston, said on May 12 during a webinar hosted by the New York Academy of Sciences called "Alienating Mars: Challenges of Space Colonization."

"That's when these kinds of technologies might be critical or necessary," she said.

Genetic enhancement may not be restricted to the pages of sci-fi novels for much longer. For example, scientists have already inserted genes from tardigrades tiny, adorable and famously tough animals that can survive the vacuum of space into human cells in the laboratory. The engineered cells exhibited a greater resistance to radiation than their normal counterparts, said fellow webinar participant Christopher Mason, a geneticist at Weill Cornell Medicine, the medical school of Cornell University in New York City.

NASA and other space agencies already take measures to protect their astronauts physically, via spacecraft shielding, and pharmacologically via a variety of medicines. So, it's not a huge conceptual leap to consider protecting them genetically as well, provided that these measures are proven to be safe, Mason said.

"And are we maybe ethically bound to do so?" he said during the webinar. "I think if it's a long enough mission, you might have to do something, assuming it's safe, which we can't say yet."

Tardigrades and "extremophile" microbes, such as the radiation-resistant bacterium Deinococcus radiodurans, "are a great, basically natural reservoir of amazing traits and talents in biology," added Mason, who has been studying the effects of long-term spaceflight on NASA astronaut Scott Kelly. (Kelly spent nearly a year aboard the International Space Station in 2015 and 2016.) "Maybe we use some of them."

Harnessing these traits might also someday allow astronauts to journey farther than Mars, out to some even more exotic and dangerous cosmic locales. For instance, a crewed journey to the Jupiter moon Europa, which harbors a huge ocean beneath its icy shell, is out of the question at the moment. In addition to being very cold, Europa lies in the heart of Jupiter's powerful radiation belts.

"If we ever get there, those are the cases where the human body would be almost completely fried by the amount of radiation," Mason said. "There, it would be certain death unless you did something, including every kind of shielding you could possibly provide."

Genetic engineering at least lets us consider the possibility of sending astronauts to Europa, which is widely regarded as one of the solar system's best bets to harbor alien life. (The Jovian satellite is a high priority for NASA's robotic program of planetary exploration. In the mid-2020s, the agency will launch a mission called Europa Clipper, which will assess the moon's habitability during dozens of flybys. And Congress has ordered NASA to develop a robotic Europa lander as well, though this remains a concept mission at the moment.)

Related: The 6 most likely places to find alien life

Genetic engineering almost certainly won't be restricted to pioneering astronauts and colonists. Recent advances in synthetic biology herald a future in which "designer microbes" help colonists establish a foothold on the Red Planet, Lynch said.

"These are some of the things that we can actually do to help us make things we need, help us make materials to build our habitats," she said. "And these are a lot of things that scientists are researching right now to create these kinds of things for our trip to Mars."

Some researchers and exploration advocates have even suggested using designer microbes to terraform Mars, turning it into a world much more comfortable for humans. This possibility obviously raises big ethical questions, especially considering that Mars may have hosted life in the ancient past and might still host it today, in subsurface lakes or aquifers. (Permanently changing our own genomes for radiation protection or any other reason may also strike some folks as ethically dubious, of course.)

Most astrobiologists argue against terraforming Mars, stressing that we don't want to snuff out or fundamentally alter a native ecosystem that may have arisen on the Red Planet. That would be both unethical and unscientific, Lynch said.

After all, she said, one of the main reasons we're exploring Mars is to determine if Earth is the only world to host life.

"And how can we do that if we go and change the planet before we go and find out if life actually was living there?" Lynch said.

Mike Wall is the author of "Out There" (Grand Central Publishing, 2018; illustrated by Karl Tate), a book about the search for alien life. Follow him on Twitter @michaeldwall. Follow us on Twitter @Spacedotcom or Facebook.

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Colonizing Mars may require humanity to tweak its DNA - Space.com

Complement genes add to sex-based vulnerability in lupus and schizophrenia – Newswise

Newswise BIRMINGHAM, Ala. Variants in a gene of the human immune system cause men and women to have different vulnerabilities to the autoimmune diseases lupus and Sjgrens syndrome, according to findings published in the journal Nature. This extends recent work that showed the gene variants could increase risk for schizophrenia.

The gene variants are a member of the complement system, a cascade of proteins that help antibodies and phagocytic cells remove damaged cells of a persons own body, as well as an infection defense that promotes inflammation and attacks pathogens. Normally the complement system keeps a person healthy in the face of pathogens; it also helps cart away the debris of damaged human cells before the body can mount an autoimmune attack. Now complement gene variants apparently play a contributing role in the diseases systemic lupus erythematosus, Sjgrens syndrome and schizophrenia.

It had been known that all three illnesses had common genetic associations with a section of the human chromosome called the major histocompatibility complex, or MHC. This region on chromosome 6 includes many genes that regulate the immune system. However, making an association with a specific gene or with the mutational variants of a specific gene that are called alleles has been difficult, partly because the MHC on human chromosome 6 spans three million base-pairs of DNA.

The Nature paper is a collaboration of 22 authors at 10 institutions in the United States and one in England, along with many members of a schizophrenia working group. Robert Kimberly, M.D., professor of medicine at the University of Alabama at Birmingham and director of the UAB Center for Clinical and Translational Science, is a co-author of the research, which was led by corresponding author Steven McCarroll, Ph.D., assistant professor of genetics at Harvard Medical School.

The identified alleles are complement component 4A and 4B, known as C4A and C4B.

The research showed that different combinations of C4A and C4B copy numbers generate a sevenfold variation in risk for lupus and 16-fold variation in risk for Sjgrens syndrome among people with common C4 genotypes. Paradoxically, the same C4 alleles that previously were shown to increase risk for schizophrenia had a different impact for lupus and Sjgrens syndrome they greatly reduced risk in those diseases. In all three illnesses, the C4 alleles acted more strongly in men than in women.

For the complement proteins that are encoded by the genes for C4 and for complement component 3, or C3, both C4 protein and its effector C3 protein were present at greater levels in men than in women in cerebrospinal fluid and blood plasma among adults ages 20-50. Intriguingly, that is the age range when the three diseases differentially affect men and women for unknown reasons. Lupus and Sjgrens syndrome affect women of childbearing age nine times more than they do men of similar age. In contrast, in schizophrenia, women exhibit less severe symptoms, more frequent remission of symptoms, lower relapse rates and lower overall incidence than men, who are affected more frequently and more severely.

Both men and women have an age-dependent elevation of C4 and C3 protein levels in blood plasma. In men, this occurs early in adulthood, ages 20-30. In women, the elevation is closer to menopause, ages 40-50. Thus, differences in complement protein levels in men and women occur mostly during the reproductive years, ages 20-50.

The researchers say sex differences in complement protein levels may help explain the larger effects of C4 alleles in men, the greater risk of women for lupus and Sjgrens, and the greater vulnerability of men for schizophrenia.

The ages of pronounced sex differences in complement levels correspond to the ages when men and women differ in disease incidence. In schizophrenia cases, men outnumber women in early adulthood; but that disparity of onset lessens after age 40. In lupus, female cases greatly outnumber male cases during childbearing years; but that difference is much less for disease onset after age 50 or during childhood. In Sjgrens syndrome, women are more vulnerable than are men before age 50.

The researchers say the differing effect of C4 alleles in schizophrenia versus lupus and Sjgrens syndrome will be important to consider in any therapeutic effort to engage the complement system. They also said, Why and how biology has come to create this sexual dimorphism in the complement system in humans presents interesting questions for immune and evolutionary biology.

Co-authors with McCarroll and Kimberly for the paper, Complement genes contribute sex-biased vulnerability in diverse illnesses, are Nolan Kamitaki, Aswin Sekar, Heather de Rivera, Katherine Tooley and Christine Seidman, Harvard Medical School, Massachusetts; Robert Handsaker and Christopher Whelan, Broad Institute of Massachusetts Institute of Technology; David Morris, Philip Tombleson and Timothy Vyse, Kings College London, London, United Kingdom; Kimberly Taylor and Lindsey Criswell, University of California-San Francisco School of Medicine; Loes Olde Loohuis and Roel Ophoff, University of California-Los Angeles; Michael Boehnke, University of Michigan; Kenneth Kaufman and John Harley, Cincinnati Childrens Hospital Medical Center, Ohio; Carl Langefeld, Wake Forest School of Medicine, North Carolina; Michele Pato and Carlos Pato, State University of New York, Downstate Medical Center; and Robert Graham, Genentech Inc., South San Francisco, California.

Support came from National Institutes of Health grants HG006855, MH112491, MH105641 and MH105653; and from the Stanley Center for Psychiatric Research.

At UAB, Kimberly holds the Howard L. Holley Research Chair in Rheumatology.

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Complement genes add to sex-based vulnerability in lupus and schizophrenia - Newswise

23andMe Is Trying to Crack the Genetic Code Behind the Coronavirus – Motley Fool

The coronavirus has swept across the globe in recent months, and companies in all sectors of the healthcare industry have stepped up to combat the pandemic. Many are now scrambling to develop a vaccine.

One of the greatest challenges facing healthcare workers on the front lines, as well as the companies working on vaccine candidates, is that COVID-19 varies so much among patients. According to a report published by the World Health Organization back in March, approximately 80 percent of those infected exhibit mild symptoms or none at all.A study published in the journal Nature Medicine found that 44% of individuals who caught COVID-19 through secondary transmission contracted the illness while the original infector was still asymptomatic.

The most recent data released from the Centers for Disease Control and Prevention shows that 60.5 per 100,000 patients require hospitalization, and death certificates show that the flu, COVID-19, or pneumonia were behind 12.8% of all U.S. deathsfrom May 4 through May 10.Over 300,000 people worldwide have died of COVID-19.

Image source: Getty Images.

DNA testing companies are rising to the occasion with the launch of numerous studies hoping to crack the genetic code behind COVID-19. Last month, one of the biggest names in the consumer genetics market, 23andMe, initiated a study into the link between genetics and COVID-19 outcomes.

Founded in 2006, 23andMe is a privately held company that offers a slew of popular direct-to-consumer genetic tests and has the power of nearly $800 million of venture capital backing behind it. While 23andMe hasn't yet made its way to the public market, prospective investors and consumers alike should still be watching this start-up and its current study closely.

Here's what you need to know.

23andMe first launched its study back in April, and hundreds of thousands of customers have participated so far through online surveys. In the initial part of its study, 23andMe surveyed customers who had tested positive for COVID-19, as well as customers who had not received a positive diagnosis.The study will also allow contributions from family members of customers who have been ill with the coronavirus. The company will use this information to assess genetic similarities and dissimilarities among critically ill patients.

Last week, 23andMe announced that it was expanding its genetic study to include up to 10,000 adults who are not 23andMe customers and who have been hospitalized for COVID-19. In 23andMe's blog post announcing the study's expansion, the company stated that its research model means it can

not only reach out to current customers, but also quickly recruit and genotype new research participants. We can then survey those participants, and conduct genetic studies at a massive scale. ... There are several questions about whether genetics may explain the differences in immune response among patients. Our study could aid in assessing differences in risk among individuals. It could also guide efforts now under way to treat the disease caused by the virus.

When 23andMe announced its expansion of the study in a blog post published May 13, it noted that more than 500,000 people have enrolled in the study thus far, and more than 7,000 of the participants have been diagnosed with COVID-19.

In the 14 years since its founding, 23andMe has received venture-capital backing from a particularly impressive and diverse group of leading technology and pharmaceutical companies. Think big names like Fidelity Management & Research Company, Sequoia Capital, Alphabet's (NASDAQ:GOOG) Google Ventures, Johnson & Johnson's(NYSE:JNJ)venture capital division, and GlaxoSmithKline (NYSE:GSK).

23andMe is in late-stage funding. Its most recent round, in January 2019, consisted of secondary market funding from angel investor and former U.S. government official Raj Luhar. Secondary market funding occurs when an individual investor buys equity in a privately held company from current investors.

There's long been talk of 23andMe entering an initial public offering. It's possible the company could be looking to secure more late-stage funding to build its momentum before going the IPO route. A 2019 report released by private investment management firm Wellington Management Company found that

companies are staying private for longer. For example, the average age to IPO for VC-backed companies increased from 4.6 years during the 1990-2001 period to 6.4 years during the 2002-2018 period. As a result, companies are often more mature when they do go public. Businesses with market capitalization below US$1 billion have decreased as a percentage of the public market, from 53% of the Russell 2000 Index in 2005 to 23% as of 31 December 2018. Amid these changes, we recognized that the late-stage growth companies were often in need of capital to accelerate growth prior to, or in lieu of, an IPO or sale.

Many thought 23andMe would IPO back in 2015 when it secured an additional $115 million in a funding round. The company has yet to announce any definite plans to go public.

It's no secret that the consumer genetics market has faced notable headwinds over the past few years, as demand for DNA testing has dropped significantly. The reason behind this decline is not certain. It could be that the early boom in consumer interest wore off, or that privacy concerns got the better of the wider market.

In January, 23andMe laid off 14% of its employees. The company was just one in a long line of venture capital-backed entities that have had to slash their workforces in recent months. For example, one of 23andMe's top competitors, Ancestry, laid off 6% of its employees in early February. Coincidentally, Ancestry has also launched a study into the genomic aspects of COVID-19, but at present is limiting participants to existing customers only.

Naturally, there have been concerns about the fiscal future of genetic services given recent reductions in consumer demand. But with this new foray into genetic research, companies like 23andMe could be well positioned to aid medical providers and businesses in establishing new protocols both during and after the coronavirus crisis. Even though the company's IPO remains uncertain, 23andMe has solid backing and could be making some big moves in the near future. Healthcare investors should find this startup definitely worth watching over the next few years.

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23andMe Is Trying to Crack the Genetic Code Behind the Coronavirus - Motley Fool

Global Molecular Diagnostics Industry 2019-2029: Genetic Disorders, Cardiovascular Disorders, Infections and Cancer – Yahoo Finance UK

Dublin, May 19, 2020 (GLOBE NEWSWIRE) -- The "Molecular Diagnostics - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

This report describes and evaluates the molecular diagnostics technologies that will play an important role in the practice of medicine, public health, pharmaceutical industry, forensics and biological warfare in the 21st century. This includes several polymerase chain reaction (PCR)-based technologies, fluorescent in situ hybridization (FISH), peptide nucleic acids (PNA), electrochemical detection of DNA, sequencing, mitochondrial DNA, biochips, nanotechnology and proteomic technologies.

The markets for molecular diagnostics technologies are difficult to estimate. Molecular diagnostics markets overlap with markets for non-molecular diagnostic technologies in the in vitro diagnostic market and are less well defined than those for pharmaceuticals.

Molecular diagnostic markets are analyzed for 2019 according to technologies, applications and geographical regions. Forecasts are made up to 2029. A major portion of the molecular diagnostic market can be attributed to advances in genomics and proteomics. Biochip and nanobiotechnology are expected to make a significant contribution to the growth of molecular diagnostics.

The number of companies involved in molecular diagnostics has increased remarkably during the past few years. More than 1,000 companies have been identified to be involved in developing molecular diagnostics and 268 of these are profiled in the report along with tabulation of 657 collaborations. Despite the strict regulation, most of the development in molecular diagnostics has taken place in the United States, which has the largest number of companies.

Initial applications of molecular diagnostics were mostly for infections but are now increasing in the areas of genetic disorders, preimplantation screening and cancer. Genetic screening tests, despite some restrictions, is a promising area for future expansion of in vitro diagnostic market. Molecular diagnostics is being combined with therapeutics and forms an important component of integrated healthcare.

Molecular diagnostic technologies are also involved in the development of personalized medicine based on pharmacogenetics and pharmacogenomics. Currently, there has been a considerable interest in developing rapid diagnostic methods for point-of-care and biowarfare agents such as anthrax. Molecular diagnostic tests for COVID-19 are described

This was the first commercial report on this topic and published as "DNA Diagnostics" in 1995 by PJB Publications, UK. A new edition in 1997 "Molecular Diagnostics I" as well as the next edition, "Molecular Diagnostics II" in 1999, were published by Decision Resources Inc, USA. All the three versions of the reports were well received and sold widely. The report has been rewritten several times.

Key Topics Covered

Part I: Technologies & ApplicationsExecutive Summary1. Introduction2. Molecular Diagnostic Technologies3. Biochips, Biosensors and Nanobiotechnology4. Proteomic Technologies for Molecular Diagnostics5. Molecular Diagnosis of Genetic Disorders6. Molecular Diagnosis of Cardiovascular Disorders7. Molecular Diagnosis of Infections8. Molecular Diagnosis of Cancer9. Molecular Diagnostics in Biopharmaceutical Industry & Healthcare10. Molecular Diagnostics in Forensic Medicine and Biological Warfare11. References

Part II: Regulations, Markets and Companies12. Ethics, Patents and Regulatory Issues13. Markets for Molecular Diagnostics14. Companies Involved in Molecular Diagnostics

For more information about this report visit https://www.researchandmarkets.com/r/4envz6

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Molecular Diagnostics Industry 2019-2029: Genetic Disorders, Cardiovascular Disorders, Infections and Cancer - Yahoo Finance UK

Prominent Cancer Researcher to Join DRI and Renown Health – GlobeNewswire

The Desert Research Institute (DRI) and Renown Health proudly announce the addition of Dr. Pier Paolo Pandolfi, MD, PhD, FRCP to the DRIs faculty of the Renown Institute of Health Innovation and as Director of the Institute of Cancer at Renown Health.

Reno, Nevada, May 18, 2020 (GLOBE NEWSWIRE) -- Reno, Nev. (May 18, 2020) Today, the Desert Research Institute (DRI) and Renown Health proudly announce the addition of Dr. Pier Paolo Pandolfi, MD, PhD, FRCP to the DRIs faculty of the Renown Institute of Health Innovation and as Director of the Institute of Cancer at Renown Health.

Dr. Pandolfi, a prominent cancer investigator and molecular geneticist, will build a translational cancer laboratory at DRIs campus in Reno, Nevada to expand the success of the Healthy Nevada Project (the largest, community-based population health study combining genetic, clinical, environmental and social data, and offering free genetic testing to every Nevadan) into translational medicine and create a world-class cancer research and clinical care program.

Dr. Pandolfi will divide his time between Reno and Italy, also leading a cancer research institute in his home country that will foster knowledge exchange and international cancer research collaborations between Italy and Nevada.

As a cancer researcher, my mission is to cure cancer. The Healthy Nevada Project and the combined resources of Renown Health and DRI give us access to an unprecedented amount of longitudinal data and the valuable genetic information we need to continue to improve our understanding of the molecular mechanisms of cancer and tailor approaches for treatments and cures that are unique to each individual said Dr. Pandolfi.

I am proud to take the unique resource of the Healthy Nevada Project, and use the information to accelerate our work to provide a population-level view of those factors that drive cancer, build better models and perhaps, timely new treatments. I am excited to build a strong collaborative bridge between the state of Nevada with our colleagues in Italy and across Europe, which will allow for the exchange of research fellows, physicians, scientists, and interns, added Pandolfi.

Dr. Pandolfi, a scientist whom the NIH deems outstanding, and who is leading significant contributions toward the understanding of cancer and genetics, is formerly the director of the cancer center at Beth Israel Deaconess Medical Center at Harvard Medical School in Boston and prior to that at Memorial Sloan Kettering Cancer Center in New York.

His extraordinary career in the molecular understanding of cancer has resulted in major medical breakthroughs in the treatment of solid tumors and leukemia. His foundational work in the study of critical cancer genes as models for tumor suppression has helped explain the causes of acute promyelocytic leukemia (APL) and led to the development of innovative and effective treatments and therapies for the disease.

Recognizing the need to expand the Healthy Nevada Project into a new era of translational medicine, we are very excited to welcome Dr. Pandolfi and his pioneering scientific bench-to-patient bedside approach, said Anthony Slonim, M.D., Dr.PH., FACHE, president and CEO of Renown Health and co-founder of the Renown Institute for Health Innovation and the Healthy Nevada Project. Dr. Pandolfis arrival in Nevada represents a significant milestone for all of us, especially those of us who are cancer survivors. Nearly 4 in 10 of us will be diagnosed with cancer, the second-leading cause of death in the US. Dr. Pandolfi understands how genomics provides new tools for the prevention and early detection of many cancers.

Through the Healthy Nevada Project, 50,000 Nevadans volunteered their genetic information. Dr. Pandolfi will use the insights gained during the first two phases of the Healthy Nevada Project to plan future research.

Dr. Pandolfi brings with him to Nevada, a prestigious National Institutes of Health (NIH), National Cancer Institute Outstanding Investigator award. This grant provides stable, long-term research funding to support the research activities of the Renown Institute of Health Innovation.

Dr. Pandolfi will also serve as Director of the Renown Institute for Cancer and further a goal to bring world-class, exceptional cancer care to Nevada. He will lead efforts to streamline, standardize, and personalize relationships at every point in the cancer care continuum screening, diagnosis, treatment, and the care provided for survivors as well as those at the end of life. In addition, Dr. Pandolfis strong connections with the research community facilitate matching Renown patients to the right clinical trials, another example of Renowns position at the leading edge of treatment while developing the cancer care of the future.

The study of human health and its connection to our environment has always had a place in DRIs mission and research activity, said Kumud Acharya, Ph.D., Interim President of DRI. We are proud to welcome Dr. Pandolfi to Nevada and we are thankful for this extraordinary opportunity to meaningfully expand our health sciences research capacity to serve Nevada, together with our partners at Renown Health.

A native of Rome, Dr. Pandolfi received his MD in 1989 and Ph.D. in 1995, both from the University of Perugia, Italy. He completed his post-graduate work at the Royal Postgraduate Medical School, University of London, before joining the faculty of Memorial Sloan-Kettering Cancer Center and the Weill Graduate School of Medical Sciences at Cornell University in New York in 1994.

He is the author of more than 450 peer-reviewed research papers and the recipient of more than 30 awards and honors, including the Leukemia and Lymphoma Society of America Stohlman Scholar Award; the Weizmann Institute of Science: Sergio Lombroso Prize for Cancer Research; the William and Linda Steere Foundation Award; and the prize for Scientific Excellence in Medicine from the American-Italian Cancer Foundation. He has also been awarded the Fondazione Cortese International Award; the Prostate Cancer Foundation Creativity Award; and the Guido Venosta Award for Cancer Research.

In 2006, Dr. Pandolfi was elected as a member of the American Society for Clinical Investigation and the American Association of Physicians and in 2007 became a member of the European Molecular Biology Organization. In 2015, Dr. Pandolfi was Knighted by the Republic of Italy, receiving the Officer of the Order of the Star of Italy by the President of the Italian Republic. More recently, Dr. Pandolfi has been elected Fellow of the American Association for the Advancement of Science (AAAS) in 2017 and Fellow of the Royal College of Physicians of London in 2018.

For more about the Renown Institute for Health Innovation at DRI please visit http://www.dri.edu/renown-ihi/.

The Renown Institute for Health Innovation is a collaboration between Renown Health a locally governed and locally owned, not-for-profit integrated healthcare network serving Nevada, Lake Tahoe, and northeast California; and the Desert Research Institute a recognized world leader in investigating the effects of natural and human-induced environmental change and advancing technologies aimed at assessing a changing planet. Renown IHI research teams are focused on integrating personal healthcare and environmental data with socioeconomic determinants to help Nevada address some of its most complex environmental health problems; while simultaneously expanding the states access to leading-edge clinical trials and fostering new connections with biotechnology and pharmaceutical companies.

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Prominent Cancer Researcher to Join DRI and Renown Health - GlobeNewswire

Research Roundup: HIV vaccination, diabetes two-in-one injection, hybrid fish genetics – The Stanford Daily

Each week, The Dailys Science & Tech section produces a roundup of the most exciting and influential research happening on campus or otherwise related to Stanford. Heres our digest for the week of May 1016.

A new vaccine type to prevent HIV infections

A new vaccination can provide enhanced and sustained protection against the HIV virus in rhesus macaque monkeys, a study published on May 11 in Nature Medicine found. The research might also help immunologists create a vaccine against the coronavirus and other diseases.

Most vaccines aim at stimulating serum immunity by raising antibodies to the invading pathogen, Bali Pulendran, professor of pathology and microbiology and immunology, told Stanford Medicine News. This vaccine also boosted cellular immunity, the mustering of an army of immune cells that chase down cells infected by the pathogen. We created a synergy between these two kinds of immune activity.

The adaptive immune response consists of two parts: serum immunity, including B-cells which secrete antibodies, and cellular immunity, including T-cells that find infected bodily cells and destroy them. The findings suggest vaccinations that stimulate both arms of the adaptive immune response can protect rhesus macaques from initial viral HIV infection.

These results suggest that future vaccination efforts should focus on strategies that elicit both cellular and neutralizing-antibody response, which might provide superior protection against not only HIV but other pathogens such as tuberculosis, malaria, the hepatitis C virus, influenza and the pandemic coronavirus strain as well, Pulendran told Stanford Medicine News.

Combination shot of insulin and amylin for diabetics

A combined two-in-one injection consisting of insulin and amylin may help diabetics better control their blood sugar levels, a study published on May 11 in Nature Biomedical Engineering found.

Previously, insulin and amylin a hormone that works with insulin to lower blood sugar levels more effectively than insulin alone could only be injected in two separate shots. Patients who have taken both drugs separately lose weight and have better control over their blood sugar levels. When combined, the drugs were too unstable for a single syringe.

Taking that second injection with the insulin shot is a real barrier for most patients, materials science and engineering assistant professor Eric Appel told Stanford News. Our formulation would allow them to be given together in a single injection or in an insulin pump.

The researchers developed a protective coating called cucurbituril-PEG that surrounds the insulin and amylin, allowing both to coexist in a single shot. The findings suggest the coating increases stability, promoting the drug shelf life.

Were excited about the results to say the least, Appel told Stanford News.

Genetic evolution of hybrid populations

Scientists have identified the cause of melanoma in hybrid fish in Mexico, a study published on May 14 in Science reports.

The highland swordtails and sheepshead swordtails have interbred for many generations and are native to Mexico, creating a population of hybrids. Researchers have identified two genes responsible for melanoma, which often develops in the tails of the male fish.

This discovery marks only the second time a dysfunction in hybrids has been traced to a specific gene in vertebrates. Hybrid offspring of two different species typically have genetic shortcomings.

Were just realizing that hybridization affects species all across the tree of life, including our own species many of us carry genes from Neanderthals and Denisovans, biology assistant professor Molly Schumer told Stanford News. Understanding hybridization and the negative and positive effects that can come from genes that have moved between species is important in understanding our own genomes and those of other species with which we interact.

The findings suggest the genes cd97 and xmrk are responsible for causing melanoma in the fish hybrids.

When I started my PhD in 2011, it was really not accepted that hybridization was common in animals. The best-known examples were mules and fruit flies. Its been such a massive shift and a fun time to be working on this question, Schumer told Stanford News. What weve arrived at now is the best kind of project in science: one that raises way more questions than answers and spins you off in a bunch of different directions.

Contact Derek Chen at derekc8 at stanford.edu.

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Research Roundup: HIV vaccination, diabetes two-in-one injection, hybrid fish genetics - The Stanford Daily

Grant will help scientists break new ground in gene editing – Newswise

Newswise AMES, Iowa A new grant will help Iowa State University researchers develop innovative gene editing technology to better understand how human genetics affect susceptibility to disease.

The four-year, $2.8 million grant from the National Institutes of Health allows Maura McGrail, an associate professor of genetics, development and cell biology and principal investigator, and her collaborators to continue their efforts to develop gene editing technologies to model human disease in zebrafish. The research aims to build new tools to determine which genes have therapeutic potential to treat human genetic diseases that affect the cardiovascular, immune and nervous systems. Zebrafish share much of their genetic makeup with humans, and the researchers hope their work will lead to more effective treatments for many of the most pressing diseases in humans.

The research team also includes Jeffrey Essner, professor of genetics, development and cell biology, and Iddo Friedberg, associate professor of veterinary microbiology and preventive medicine.

The technology that weve developed allows us to modify genes in a precise way that places gene activity under spatial and temporal control, McGrail said. One of the challenges in disease research is understanding the impact of gene loss at the cellular level. By modeling genetic disease in zebrafish, were able to reconstitute the complexity of human disease in a living system and visualize how cell processes are disrupted.

The method developed by McGrail and her colleagues combines gene editing technology with site-specific recombination enzymes. Utilizing the two in concert allows the scientists to control where and when a gene is deactivated. The new grant will also test approaches to optimize the efficiency of their method and expand on their ability to introduce more subtle gene changes frequently found in human disease genes.

Its like flipping a switch, McGrail said. We can deactivate a gene in a specific tissue, and investigate how that affects cell viability and organ function, providing insight into the process of disease pathology.

The research team also published an article in the peer-reviewed academic journal eLife, released this week, that lays the foundation for much of what they will do with the new grant. The paper describes the researchers ability to use gene editing to target integration into the genome, or introduce new DNA into the genome with great precision.

The zebrafish is a small, freshwater species, usually only a few centimeters in length, that makes an ideal model organism for genetic study because of the species transparent embryos, which allow for easy study. The zebrafish genome also shares up to 80% of the genes that cause disease in humans, Essner said.

Because of that high degree of conservation, we believe we can reach many human diseases, he said.

For instance, the researchers foresee a future in which gene editing technology could be used to treat certain kinds of cancer. McGrail said disease progression in some cancers is caused by gene deactivation, but what if doctors could reactivate those genes? Would that suppress the growth of tumors? McGrail and Essner said their research will take an important step toward answering such questions.

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Grant will help scientists break new ground in gene editing - Newswise