Just because you've watched Jurassic Park doesn't mean you know that gene editing is bad

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Gene editing is an important area for further research.

Gene editing is the future and we should embrace it. I dont mean a wholehearted approval of the technique but to recognize that its here. We must be thoughtful about its applications and aware of its potential.

Weve been gene editing all throughout history. Selectively breeding animals and crops to promote the traits that are desirable or helpful to us. But todays gene editing is much different.

We use CRISPR-Cas9, which can target specific gene sequences to edit, said Samantha Noll, a bioethicist with the Functional Genomics Initiative.

CRISPR, a gene editing tool taken from bacterial defenses against viruses, allows molecular biologists here at WSU to alter specific genes in big animals. Compared to CRISPR, selective breeding is crude and inaccurate, only using phenotypic traits, such as eye or hair color, as the roadmap for which animal to breed or not. Selective breeding attempts to manipulate the genome by prioritizing expressed traits whereas CRISPR allows the manipulation of the genome by access to the entire gene pool.

Charlie Powell, the public information officer for WSUs school of Veterinary Medicine, mentioned multiple ways this gene editing technique can be applied positively.

At any given moment here in the US there are a million pigs in transit. A certain percentage of those animals will develop upper respiratory diseases as a result of the stress, Powell said. If we could make those pigs resistant instead of vaccinating them then we have the possibility of limiting those losses in the industry. This involves adding back the wild-type genes that they originated with.

This suggests ethical solutions by way of medical intervention. Lingering just on livestock application, how much animal suffering could be eliminated by well-applied selective gene editing? Instead of injecting tons of antibiotics, genetic immunity may be the way to go.

Fostering the path to healthier and happier livestock could be inroads to alleviating human challenges such as hunger and poverty. Abundance of sustainable and ethically produced meats could ease food demand, and resilient healthy livestock could be a valuable investment for underprivileged individuals.

Being a land-grant university, WSU research is primarily aimed at helping the local community, hence the focus on big animals and local agriculture. One of the research programs seeks to knock out the genes responsible for horns in cattle. This avoids the painful horn removal process for the animals and prevents accidental injury between cattle, which cost time and money.

Though these are promising initiatives, we cant be short-sighted either. William Kabasenche, a bioethicist focusing on the therapeutic applications of CRISPR, described what he called off-target effects.

Its called pleiotropy, when one gene influences multiple phenotypes, Kabasenche said.

Phenotypes are just the expressed traits. The information for those traits is stored in the gene. Off-target effects occur when a gene has unaccounted phenotypes, meaning that the manipulation of that gene produced an unforeseen or undesirable change in a phenotype.

This is why we have to be very careful when gene editing. Yes, the potential is huge both for scientific discovery as well as the well-being of conscious entities but we must guard against a utopic vision of the technology. There are trade-offs involved. Changing one gene may produce the desired effects, but drastically impact an unrelated but necessary function.

This stresses the need for research and the role of ethics in research. We should all want this work to be done, but we cannot simply focus on positive outcomes and draw the conclusion that it justifies its good. We must also consider how these outcomes are achieved.

We must also consider the harmful potential of gene editing. How we choose to engage our resources, the decisions and norms we set in research will in some part determine how well apply this technology. These norms are being born at research institutions like WSU.

Its a promising start that WSU includes ethicists, educators and biologists to tackle these difficult issues.

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OPINION: Support gene researchers - The Daily Evergreen

A team of researchers led by Dr Hugo Bellen at Baylor College of Medicine, investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Childrens Hospital and also a Howard Hughes Medical Institute investigator, and lead author, Hyunglok Chung, postdoctoral fellow in the Bellen lab, have discovered that a mutation in a gene that causes Mitchell disease.

However, they also discovered that the gene was not inherited, but was in fact a new mutation.

A patient with neurological symptoms enrolled in the Undiagnosed Diseases Network (UDN), presenting with an unidentified late-onset neurodegenerative disorder.

Bellen said: On comparing the patients and his parents DNA, the team identified a mutation in the patient that resulted in a single amino acid substitution (N237S) in the ACOX1 protein. This change was seen only in the patient and was not present in either of his parents DNA, indicating that the patient had a de novo, or new, mutation on this gene

With the help of the online gene-matching tool GeneMatcher, we found two more patients who had the same new mutation in the ACOX1 gene.

All three patients, who ranged from 3 to 12 years old at the time of disease onset, had remarkably similar clinical features, including degeneration of peripheral nerves that caused a progressive loss of mobility and hearing. The three individuals had identical gene variants, a clear indication that ACOX1 dysfunction likely was the cause of the symptoms.

The finding that an ACOX1 mutation was linked to Mitchell disease initially baffled the researchers. The only known ACOX1-related disorder described in the medical literature at that time presented earlier in infancy with seizures, severe cognitive decline, neuro-inflammation and accumulation of very-long-chain-fatty acids in plasma and, more importantly, was caused by the lack of the ACOX1 protein none of which was true for these three patients.

The brain has large amounts of lipids, which are critical for the proper functioning of the nervous system. Abnormal breakdown of lipids in the brain and peripheral nervous system is associated with several neurodegenerative diseases, Bellen said.

The gene ACOX1 is involved in lipid breakdown. It produces an enzyme called Acyl-CoA oxidase 1 that initiates a series of reactions that break down very-long-chain-fatty acids in small intracellular organelles called peroxisomes.

The team used fruit flies to understand the problem, with Chung discovering that the ACOX1 protein is abundant and critical for the maintenance of glia, cells that support neurons.

To gain a better understanding of how ACOX1 variants affect the function of glia, they generated two mutant fly lines, the first one lacked both the copies of ACOX1 gene and the second, carried the substitution mutation (N237S) found in one of the ACOX1 genes in the Mitchell disease patients.

Chung said: Flies lacking ACOX1 mimicked the symptoms of ACOX1 deficiency in humans, including elevated levels of very-long-chain-fatty acids along with dramatic loss of glia and neurons and progressively impaired neuronal function. When we reduced the synthesis of very-long-chain-fatty acids in these flies by administering the drug bezafibrate, we observed significant improvement in lifespan, vision, motor coordination and neuronal function, implicating elevated levels of these lipids and their excessive accumulation in glia as an important contributor.

The researchers suggest that bezafibrate could offer a new therapeutic avenue for patients.

In contrast to the loss of ACOX1, the introduction of the single amino acid substitution (N237S) in ACOX1 gene resulted in a hyperactive ACOX1 protein.

Typically, breakdown of very-long-chain-fatty acids by the enzymatic action of ACOX1 produces small amounts of highly reactive oxygen species, but glial cells quickly neutralise them. However, in Mitchells disease, hyperactive ACOX1 produces copious amounts of toxic reactive oxygen species, leading to the destruction of glia and their neighbouring neurons.

The harmful effects due to hyperactive ACOX1 were potently reversed with the antioxidant N-acetyl cysteine amide (NACA). However, NACA did not suppress the lethality or toxic effects in flies that lacked ACOX1, a clear indication that the two diseases act via entirely different pathways and would need to be treated with two distinct therapeutic strategies.

Bellen said: This study is a prime example of how combining UDNs unique team science approach with power of fruit fly genetics is facilitating rapid and phenomenal progress in rare diseases research. We take on cases of patients with conditions never described before, uncover new diseases, and find definitive molecular diagnosis for them. We make significant progress in unravelling the causes of these novel diseases and rapidly identify and test promising new treatment options.

We have successfully identified more than 25 disease-causing genes within the past three years a task that typically takes many years.

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Mitchell disease: solving the medical mystery - Health Europa

Easy-to-understand genetic counseling reports will lead to more appropriate follow-up and better health outcomes

NEW HAVEN, Conn., April 16, 2020 /PRNewswire-PRWeb/ -- My Gene Counsel, a digital health company that provides innovative genetic counseling solutions, today announced it is teaming up with UConn Health's Neag Comprehensive Cancer Center's Hereditary Cancer Program to ensure that UConn's patients who have undergone genetic testing for cancer predisposition have access to timely and accurate genetic counseling information through the online delivery of My Gene Counsel's Living Lab Reports. Together, My Gene Counsel and UConn Health will use state-of-the-art digital tools to efficiently communicate up-to-date genomic information that will drive an improved standard of care.

By supplementing its current genetic counseling services with My Gene Counsel's digital counseling tools, UConn Health will serve as a leader in this space with the ability to better support patients over time in the post-test period. Each incoming patient will receive an electronic Living Lab Report sponsored by UConn Health and the Linda Clemens Breast Cancer Foundation that is personalized to the individual's genetic test results and outlines the most frequently asked questions and answers on topics related to disease risk, medical management options, relevance to family, emotional support, and available resources. This information is supported by tools to enhance understanding, such as hover dictionary and graphics.

"Genetics can be overwhelming and confusing, so when a patient leaves my office, I worry about how much information they have retained," said Connor Linehan, MS, LGC, a board-certified genetic counselor for the Hereditary Cancer Program at UConn Health. "Our goal, in partnering with My Gene Counsel, is to increase patient understanding in the hopes that better comprehension equals appropriate medical follow-up and better health outcomes. The addition of a user-friendly genetic counseling report that patients can review before and after their appointment and over time will be invaluable to empower them to make informed decisions about their healthcare."

The Living Lab Reports are written and continuously updated by a network of top certified genetic counselors and medical experts and are vetted by patient advocates. In addition to delivering complex genomic information in a way that patients can understand, the reports will update and notify patients automatically by text and/or email as My Gene Counsel adds new information to reflect changes in disease risk, medical management options, variant reclassification, and relevant clinical trials.

"I encourage patients to contact us over the years as information about hereditary cancer changes over time," said Jennifer Stroop, MS, CGC, LGC, a board-certified genetic counselor for the Hereditary Cancer Program at UConn Health. "However, this is not always easy. We are very excited to now be able to offer our patients a reference tool with continuing updates and notifications. With these continued touchpoints for engagement and retention, we will be able to meet the expressed need to help our patients feel more connected and supported in the long term."

My Gene Counsel's wraparound solution, available at UConn in May 2020, will enable the responsible return of results, engage and update patients, and integrate data into UConn's health care system. Living Lab Reports will be personalized by gene and variant and provided for all results, whether testing is negative or identifies a variant of clinical or uncertain significance.

"We are excited to partner with UConn Health, a forward-thinking health system on the cutting-edge of hereditary cancer and precision medicine," said Ellen Matloff, MS, CGC, president and CEO of My Gene Counsel. "Their dedication to improving health through education, innovation, and patient-centered clinical care beyond the initial genetic test aligns seamlessly with our own ideals."

More than 600 people undergo genetic counseling and testing each year as part of the Neag Comprehensive Cancer Center's Hereditary Cancer Program, which is staffed by two genetic counselors. The expanding volume of patients and limited bandwidth led the team to proactively seek out a technical solution that could help solve the challenge of monitoring critical clinical updates and research and recontacting patients.

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"It is essential for UConn Health, as a major university center, to keep pace with the growing demand for up-to-date genomic information," said Susan Tanenbaum, MD, medical director of the Neag Comprehensive Cancer Center. "The integration with My Gene Counsel is a giant step towards UConn Health assuming a position of global leadership in genomics and personalized medicine."

About My Gene Counsel: My Gene Counsel bridges the gap between genetic testing and precision medicine by addressing one of the most critical pain points in the industry lack of accurate and timely genetic counseling information for patients and providers. Their Living Lab Reports deliver genetic counseling information that updates as new clinical information emerges, guidelines evolve, and genetic variants are reclassified. Founded by certified genetic counselors with 30+ years of clinical experience, My Gene Counsel empowers partners to efficiently deliver on the promise of precision medicine. For more information, visit http://www.mygenecounsel.com.

About UConn Health: UConn Health is Connecticut's only public academic medical center. Based on a 206-acre campus in Farmington, UConn Health has a three-part mission: research, teaching and patient care. Home to the UConn School of Medicine, School of Dental Medicine and UConn John Dempsey Hospital with nearly 5,000 employees supporting nearly 1,000 students, over 800,000 annual patient visits, and innovative scientific research contributing to the advancement of medicine. For more information, visit http://www.health.uconn.edu.

SOURCE My Gene Counsel

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My Gene Counsel Partners with UConn Health to Provide Expanded Genetic Counseling Services - Yahoo Finance

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

Another COVID-19 Vaccine Looks Promising

According to the World Health Organization, there are 70 vaccines being developed worldwide for COVID-19, with three already in human clinical trials. The furthest along is one by CanSino Biologics and the Beijing Institute of Biotechnology, which is in Phase II. The others are by U.S. companies, Inovio Pharmaceuticals and Moderna. Another institution, the University of Pittsburgh, also announced that in laboratory tests their COVID-19 vaccine, delivered via a fingertip-sized patch, showed positive results in laboratory mice, producing antibodies specific to SARS-CoV-2 at enough amounts to neutralize the virus. The research was published in EBioMedicine, published by The Lancet.

We had previous experience on SARS-CoV in 2003 and MERS-COV in 2014, said co-senior author Andrea Gambotta, associate professor of surgery at the Pitt School of Medicine. These two viruses, which are closely related to SARS-CoV-2, teach us that a particular protein, called a spike protein, is important for inducing immunity against the virus. We know exactly where to fight this new virus. Thats why its important to fund vaccine research. You never know where the next pandemic will come from.

It uses a more traditional process than the mRNA one being used by Moderna. The virus is being called PittCOVacc, and uses laboratory-manufactured pieces of viral protein to build immunity. Its a process similar to that used in seasonal flu shots. They also leveraged a new technique to deliver the drug, called a microneedle array, to increase the potency of the vaccine. The fingertip-sized patch has 400 tiny needles that deliver the spike protein pieces into the skin. It goes on like a Band-Aid. The needles are built from sugars and the antigens, and they just dissolve.

We developed this to build on the original scratch method used to deliver the smallpox vaccine to the skin, but as a high-tech version that is more efficient and reproducible patient to patient, said Louis Falo, co-senior author and professor and chair of dermatology at Pitts School of Medicine and UPMC. And its actually pretty painlessit feels kind of like Velcro.

They are currently submitting an Investigational New Drug (IND) with the U.S. Food and Drug Administration (FDA) and hope to begin Phase I human clinical trials in the next few months.

Genetic Mechanisms of Inflammatory Bowel Disease

Researchers at Children's Hospital of Philadelphia identified a genetic variant that causes the development of inflammatory bowel disease (IBD). The pathway is linked to other immune disorders. More than 240 genetic regions are already associated with IBD, but each region has multiple markers and not all are causative. The researchers focused on the single nucleotide polymorphism (SNP) rs1887428, located on the promoter region of the JAK2 gene. The protein coded by the gene controls the production of blood cells. The team found that two transcription factors, RBPJ and CUX1, recognize the DNA sequence altered by the rs1887428 SNP, and while it only has mild influence on JAK2 expression, it was amplified by other proteins in the JAK2 pathway.

Possible Mechanism of Link Between Obesity and Breast Cancer

Breast cancer (and other cancers) and obesity are associated, but the reason for that link isnt well understood. Researchers from the University of Louisville published research suggesting that the fatty acid binding protein family, especially FABP4, plays a critical role. Fat tissue produces FABP4 within fat cells, which processes and distributes water-insoluble long-chain fatty acids. Normally, some FABP4 enters the bloodstream, but the higher fat volume, the more FABP4 is secreted. They believe two mechanisms are in play. Within cells, FABP4 increases in certain tumor-associated macrophages, which accumulate in tumors and promote cancer growth. And second, when elevated levels of FABP4 circulate outside the fat cells, it promotes breast cancer by directly interacting with breast cancer cells.

Using Cellular Machinery Without the Cells to Develop Drugs

Northwestern University and ShanghaiTech University leveraged cell-free synthetic biology to produce a drug that kills SARS-CoV-2 in cell cultures. They indicate they could create the new drug by taking the molecular machinery out of cells and using that machinery to make a product in a safe, cheap and quick way. The molecule is called valinomycin. By using this method, they were able to increase production yields more than 5,000 times in only a few quick design cycles.

Glucose Metabolism Linked to Alzheimers Disease

Researchers with the National Institutes of Healths National Institute on Aging conducted the largest study so far on proteins related to Alzheimers and identified proteins and biological processes that regulate glucose metabolism that are associated with Alzheimers. The study was published in the journal Nature Medicine.

The study was part of the Accelerating Medicines Partnership for Alzheimers Disease (AMP-AD). The investigators assayed the levels and analyzed the expression patterns of more than 3,000 proteins in brain and cerebrospinal fluid samples collected at centers across the U.S.

This is an example of how the collaborative, open science platform of AMP-AD is creating a pipeline of discovery for new approaches to diagnosis, treatment and prevention of Alzheimers disease, said Richard J. Hodes, NIA director. This study exemplifies how research can be accelerated when multiple research groups share their biological samples and data resources.

The study involved analyzing protein expression patterns in more than 2,000 human brain and almost 400 cerebrospinal fluid samples taken from both healthy individuals and Alzheimers patients. They analyzed how the protein modules relate to Alzheimers and other neurodegenerative diseases. They observed changes in proteins related to glucose metabolism and an anti-inflammatory response in glial cells in brain tissues from both Alzheimers patients and people with documented brain pathology who were cognitively normal. This also would seem to support increasing evidence that brain inflammation is involved in the disease as well.

In Alzheimers patients, they found that how cells extract energy from glucose is increased in both the brains and spinal fluid of Alzheimers patients. The proteins observed were also elevated in preclinical Alzheimers patients, which is to say, people with brain pathology of the disease who had not shown cognitive decline.

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Research Roundup: Another Promising COVID-19 Vaccine and More - BioSpace

Evotec is building a gene therapy R&D center manned by ex-Takeda scientists and has already landed a multi-year drug discovery pact for it with their former Japanese employer.

Evotecis expanding into the field of gene therapy by building an R&D center in Austria using scientists previously employed by Takeda, whose first project will be a long-term research and discovery project with their former Japanese employer, exploring oncology, rare diseases, neuroscience and gastroenterology.

The entry by the German discovery alliance and development partnership group into gene therapy further expands its capabilities beyond small molecules, cellular therapies and biologics while the research alliance withTakeda Pharmaceutical, announced on 6 April, builds upon an existing collaboration begun last September around drug discovery programs in which Evotec will deliver clinical candidates for Takeda to pursue into clinical development.

Image: iStock/sittithat tangwitthayaphum

This addition of gene therapy is the latest step in a very long strategy at Evotec to build a truly comprehensive organization which can deliver medicines discovery and development right across the different modalities, or scientific tools, that we see in modern medicine today, Evotec chief operating officer Craig Johnstone toldScrip.

He said the move was consistent with Evotecs two-pronged drug discovery strategy, which uses the Hamburg-based groups so-called Execute segment allying with external pharma partners which in turn supports reinvestment into Evotecs internal pipeline within its so-called Innovate segment.

This allows us to bring gene therapy project concepts into the Evotec Innovate pipeline at our choice and discretion. That was not possible before, so we can now use gene therapy to support our partners and also use it to support ourselves.

Johnstone said Evotec already makes broad use of CRISPR in its discovery and development activities. This will only amplify that aspect of Evotecs activities, he added.

Evotec Gene Therapy (Evotec GT) will start operations with a team of gene therapy experts at an R&D site in Orth an der Donau, Austria. Its scientists have deep expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases.

We are recruiting the team, which will be composed of ex-Takeda employees. Its leadership has a long history in gene therapy stretching back years and to legacy organizationsBaxaltaandShire which were acquired by Takeda, but who were made redundant. Well be bringing them all on board over the next eight weeks, Johnstone said. Friedrich Scheiflinger, previously head of drug discovery for Takeda in Austria, will head up the new gene therapy unit.

No financial details were disclosed about Evotecs latest collaboration with Takeda.

Its a fairly straight-forwardcontractual framework that has been agreed between Evotec and Takeda, Johnstone said, without elaborating.

Sten Stovall is a London-based editor and writer with 40 years of experience in the field of journalism, including more than 20 years with Reuters and eight years with The Wall Street Journal/Dow Jones Newswires. He can be reached at sten.stovall@informa.com

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Evotec extends Takeda pact to gene therapy - Bioprocess Insider - BioProcess Insider

-Spinal cord injury (SCI) creates a complex microenvironment that is not conducive to repair; growth factors are in short supply, whereas factors that inhibit regeneration are plentiful. In a new report, researchers have developed a structural bridge material that simultaneously stimulates IL-10 and NT-3 expression using a single bi-cistronic vector to alter the microenvironment and enhance repair. The article is reported in Tissue Engineering, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the article for free on the Tissue Engineering website through May 17, 2020.

In Polycistronic Delivery of IL-10 and NT-3 Promotes Oligodendrocyte Myelination and Functional Recovery in a Mouse Spinal Cord Injury Model, Lonnie D. Shea, PhD, University of Michigan, and coauthors report the development of a new poly(lactide-co-glycolide) (PLG) bridge with an incorporated polycistronic IL-3/NT-3 lentiviral construct. This material was used to stimulate repair in a mouse SCI model. IL-10 was included to successfully stimulate a regenerative phenotype in recruited macrophages, while NT-3 was used to promote axonal survival and elongation. The combined expression was successful; axonal density and myelination were increased, and locomotor functional recovery in mice was improved.

Inflammation plays a vital role in tissue repair and regeneration, and the use of a PLG bridge to take advantage of the inflammatory response to promote SCI repair is an elegant way to take advantage of these natural processes to improve SCI healing, says Tissue Engineering Co-Editor-in-Chief Antonios G. Mikos, PhD, Louis Calder Professor at Rice University, Houston, TX.

About the Journal

Tissue Engineering is an authoritative peer-reviewed journal published monthly online and in print in three parts: Part A, the flagship journal published 24 times per year; Part B: Reviews, published bimonthly, and Part C: Methods, published 12 times per year. Led by Co-Editors-in-Chief Antonios G. Mikos, PhD, Louis Calder Professor at Rice University, Houston, TX, and John P. Fisher, PhD, Fischell Family Distinguished Professor & Department Chair, and Director of the NIH Center for Engineering Complex Tissues at the University of Maryland, the Journal brings together scientific and medical experts in the fields of biomedical engineering, material science, molecular and cellular biology, and genetic engineering. Leadership of Tissue Engineering Parts B (Reviews) and Part C (Methods) is provided by Katja Schenke-Layland, PhD, Eberhard Karls University, Tbingen, Heungsoo Shin, PhD, Hanyang University; and John A. Jansen, DDS, PhD, Radboud University, and Xiumei Wang, PhD, Tsinghua University respectively. Tissue Engineering is the official journal of the Tissue Engineering & Regenerative Medicine International Society (TERMIS). Complete tables of content and a sample issue may be viewed on the Tissue Engineering website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Stem Cells and Development, Human Gene Therapy, and Advances in Wound Care. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industrys most widely read publication worldwide. A complete list of the firms 90 journals, books, and newsmagazines is available on the e Mary Ann Liebert, Inc., publishers website.

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Co-delivery of IL-10 and NT-3 to Enhance Spinal Cord Injury Repair - Mirage News

About a month ago, Medical News Today started a series aiming to bring together the more encouraging research that emerges around COVID-19. We continue with this Special Feature that focuses on an incoming vaccine and other potential treatments for this new coronavirus and the disease it causes.

With this series, we aim to remind our readers that while COVID-19 causes great sorrow and loss around the world, the resulting global emergency has also meant that scientists are working at an unprecedented pace. They are making progress that is easy to overlook among the worrying numbers of new cases and deaths.

Two recent MNT articles COVID-19: 5 reasons to be cautiously hopeful and COVID-19: Physical distancing, drug trials offer hope looked at the latest developments in potential treatments, vaccines, and the outcomes of infection control measures during the pandemic.

We continue our series with this third Special Feature, which continues to monitor progress in the areas mentioned above.

Stay informed with live updates on the current COVID-19 outbreak and visit our coronavirus hub for more advice on prevention and treatment.

We focus on a vaccine that some researchers believe may be available by the fall and round up expert opinions on this promising development. We also cover an app-based social tracing system that could help create intelligent physical distancing instead of national lockdowns.

We previously reported that the World Health Organization (WHO) have launched a global megatrial that involves testing four potential treatments for COVID-19. Remdesivir, initially developed to treat Ebola, was one of those four potential treatments.

Now, scientists from the University of Alberta in Edmonton, Canada, say that remdesivir is showing promise in in vitro experiments.

The same team had previously demonstrated that remdesivir effectively combatted another coronavirus, MERS-CoV. It did so by blocking polymerases, which are enzymes that allow the virus to replicate.

Study co-author Prof. Matthias Gtte explains, If you target the polymerase, the virus cannot spread, so its a very logical target for treatment.

He continues to report the results of the teams new experiments: We obtained almost identical results as we reported previously with MERS, so we see that remdesivir is a very potent inhibitor for coronavirus polymerases.

Prof. Gtte goes on to explain, These coronavirus polymerases are sloppy, and they get fooled, so the inhibitor gets incorporated many times, and the virus can no longer replicate.

Still, the author cautions, Weve got to be patient and wait for the results of the randomized clinical trials.

Another hopeful finding comes from researchers from Cornell University in Ithaca, NY. These scientists also started their research efforts by drawing parallels with other coronaviruses, such as SARS-CoV and MERS-CoV.

Namely, they looked at the spike protein that coronaviruses have and zoomed in further on the fusion peptides these are short-chain amino acids that the spike proteins contain.

Whats really interesting about SARS-CoV and MERS-CoV, and this new virus, SARS-CoV-2, is this particular part of the protein, the fusion peptide, is almost exactly the same in those three viruses, explains study co-author Prof. Susan Daniel.

The new study found that calcium ions enable fusion peptides to help coronaviruses penetrate healthy cells through a process called membrane fusion. This offers a potential target for a new antiviral treatment.

The team has already secured funding to start developing an antibody that could stop this process by targeting SARS-CoV-2s fusion peptide.

Blocking the fusion step is significant because the fusion machinery doesnt evolve and change as fast as other parts of the protein do. Its been built to do a particular thing, which is to merge these two membranes together. So if you can develop antiviral strategies to reduce that efficiency, you could have potentially very broadly-acting treatments.

Prof. Susan Daniel

Sarah Gilbert, a professor of vaccinology at Oxford Universitys Jenner Institute in the United Kingdom, and her team may soon be closing in on a vaccine for SARS-CoV-2.

The approach uses a harmless chimpanzee virus to carry the fragment of SARS-CoV-2 that is required for immunity, explains Ian Jones, Professor of virology at the University of Reading, U.K.

Colin Butter, an associate professor of bioveterinary science at the University of Lincoln in the UK, explains: Professor Gilberts team [] have made a recombinant vaccine against the SARS-CoV-2 virus by taking a virus that is entirely harmless to humans, the Chimp Adenovirus designated ChAdOx1, and inserting into it the spike protein gene from the [new] coronavirus.

Prof. Gilbert believes that the vaccine will be available for general use by the fall, which could prevent a potential second wave of the new coronavirus.

That is just about possible if everything goes perfectly, Prof. Gilbert told The Times in an interview. The researchers are set to put the new vaccine into human trials in the next 2 weeks.

The researcher explains that during the pandemic, scientists can fast-track the process through which the vaccine reaches the population by doing many of the necessary steps in parallel.

First, there is the need to manufacture the vaccine for clinical studies under tightly controlled conditions, certified and qualified we need ethical approval and regulatory approval. Then, the clinical trial can start with 500 people in phase I.

This is always in healthy adults aged about 18 to 55, and usually the primary read-out from a phase 1 study is safety, Prof. Gilbert explains. Then we can do phase 2, looking at a wider age range; in this case, we are going to increase the age range, 55 to 70 plus. We are looking at safety in the older age group, [and] we expect to see weaker immune responses.

The researcher explains that she and her team plan to spread their studies across different countries so that they can reduce the time it takes to test the vaccine.

[I]ts vital we go fast before a high proportion [of the population] become infected. But it also means we are going to need to do studies in different countries because the amount of virus transmission is affected by the lockdowns.

The vaccine could get approval under emergency use legislation, meaning that in an emergency situation, if the regulators agree, its possible to use a vaccine earlier than in normal circumstances, Prof. Gilbert adds.

It is worth noting that other experts have expressed concern over Prof. Gilberts estimates.

Prof. David Salisbury, for example, says, [I]t is not just the availability of the first dose that we need to focus on. We need to know by when there will be sufficient doses to protect all of the at-risk population, probably with two doses, and that means industrial-scale manufacturing that governments do not have.

The approach in itself, however, is viable, and the research group benefits from a lot of credibility in the scientific community. The approach has been extensively tested in other situations, so there is indeed a good chance it will work as designed, says Prof. Jones.

The [research] group has a long history of success in this area, adds Dr. Butter. On the basis of this prior experience, it would be reasonable to assume that the vaccine would induce antibody and cellular immune responses, both of which may be important in controlling the virus in an individual.

Any final roll-out will almost certainly need a level of manufacturing the [U.K.] does not readily have, so transfer to and liaison with an external manufacturer may also need to be tackled. But the roadmap is clear, lets hope they get there.

Prof. Ian Jones

Tissue plasminogen activator (tPA) is a drug designed and approved to prevent blood clots in people who have had a stroke, pulmonary embolism, or heart attack.

Now, a new trial to test its benefits for relieving acute respiratory distress syndrome (ARDS) in people with COVID-19 is underway.

TPA acts as an anticoagulant. This means it prevents blood clotting by breaking down fibrin. Fibrin can form plugs in the airways and contributes to small clots in the blood vessels of the lungs.

In patients with COVID-19, these small microfibrin plugs in the air sacs lead to ARDS. As a result, these patients require ventilators to be able to breathe.

Were hearing anecdotally that a subset of patients with COVID-19-induced ARDS are clotting abnormally around their catheters and [intravenous] lines, explains Dr. Michael B. Yaffe, Ph.D., an acute care surgeon at the Beth Israel Deaconess Medical Center (BIDMC) in Boston, MA.

Dr. Yaffe is also the senior author of the study that proposed repurposing tPA to treat COVID-19 complications.

We suspect these patients with aggressive clotting will show the most benefit from tPA treatment, and this new clinical trial will reveal whether thats the case, says Dr. Yaffe.

The scientists have started to recruit some of the COVID-19 patients admitted to the BIDMC for the trial. The team also hopes to find biomarkers that can help identify patients who are most likely to benefit from the treatment.

If effective and safe for the treatment of ARDS in patients with COVID-19, tPA could save lives by reducing recovery time and freeing up more ventilators for other patients in need.

Christopher D. Barrett, clinical trial investigator

Another finding that may help relieve the pressure on public health systems is a mobile app-based contact tracing system.

The authors of the new project explain that such a system could help reduce the rate at which the virus spreads while also mitigating some harmful effects of a full national lockdown.

Dr. David Bonsall senior researcher at Oxford Universitys Nuffield Department of Medicine, clinician at Oxfords John Radcliffe Hospital both in the UK, and co-lead of the project explains how the system works.

He says, The mobile app concept weve mathematically modeled is simple and doesnt need to track your location. It uses a low energy version of Bluetooth to log a memory of all the app users with whom you have come into close proximity over the last few days.

If you then [contract the virus], these people are alerted instantly and anonymously and advised to go home and self-isolate. If app users decide to share additional data, they could support health services to identify trends and target interventions to reach those most in need.

The findings could pave the way for intelligent [physical] distancing, avoiding the social and economic effects of full lockdowns.

For live updates on the latest developments regarding the novel coronavirus and COVID-19, click here.

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COVID-19: Vaccine may be ready by fall and other reasons for hope - Medical News Today

BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics Inc. (Nasdaq: SGMO), a genomic medicine company, today announced the appointment of D. Mark McClung as Executive Vice President and Chief Business Officer. Mr. McClung will oversee commercial strategic planning, alliance management and corporate and business development.

Mr. McClungs appointment is the latest in the evolution of Sangamos leadership implemented over the last three years as the Companys technology and research programs have advanced into a diversified pipeline of therapeutic product candidates in various stages of clinical development. During this period, Sangamo has also appointed executive vice presidents overseeing R&D, manufacturing, legal and finance.

Im excited to welcome Mark to Sangamo. With our first product candidate entering Phase 3 and our broad pipeline of proprietary and partnered programs advancing in development, we are increasingly focused on late stage development and commercialization strategies for genomic medicines. Mark has extensive experience leading commercial organizations in therapeutic areas where innovative products have disrupted standards of care, said Sandy Macrae, Sangamos CEO.

From 2015 through 2019, Mr. McClung was Vice President and General Manager of Global Oncology Commercial at Amgen, which he joined from Onyx Pharmaceuticals where he had served as Chief Commercial Officer. For two decades prior, Mr. McClung held roles of increasing responsibility at GlaxoSmithKline in marketing and sales, commercial operations, and general management in the United States and Europe, including as Vice President and Head of Global Commercial for GSK Oncology from 2009 2013.

Over the next decade, genomic medicines have the potential to transform the practice of health care across therapeutic areas from rare monogenic diseases to immunology and oncology, and even to highly prevalent neurological disorders such as Alzheimers disease and Parkinsons disease, Mr. McClung commented. With its deep scientific expertise, diverse technology platforms, broad pipeline and significant collaborations, Sangamo is well positioned for this new era, and Im thrilled to join the Company at this time.

Stephane Boissel, Executive Vice President of Corporate Strategy, will leave Sangamo at the end of July and eventually return to an entrepreneurial project. Mr. Boissel joined Sangamo in 2018 following the acquisition of TxCell (now Sangamo France), where he had served as CEO.

Stephanes impactful contributions to Sangamo will endure for many years. He has driven several remarkable deals to fruition, including most recently our transaction with Biogen, which is among the largest preclinical collaboration deals ever, Macrae said. It has been an enormous pleasure working with Stephane these last two years, and we wish him every success in the future.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Sangamo Forward Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential to develop, obtain regulatory approvals for and commercialize immunology and oncology therapies, therapies to treat rare monogenic diseases, neurological diseases and other diseases and other therapies and the timing and availability of such therapies, the potential for Sangamo to receive upfront licensing fees and earn milestone payments and royalties under the Biogen and other collaborations and the timing of such fees, payments and royalties, Sangamos product pipeline, technology platforms and scientific expertise, Sangamos financial resources and expectations and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the research and development process; the regulatory approval process for product candidates; the commercialization of approved products; the potential for technological developments that obviate Sangamo's technologies; the potential for Biogen to breach or terminate the collaboration agreement; and the potential for Sangamo to fail to realize its expected benefits of the Biogen and other collaborations. There can be no assurance that Sangamo will earn any upfront licensing fees or milestone or royalty payments under the Biogen or other collaborations or obtain regulatory approvals for product candidates arising from these collaborations. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's filings with the U.S. Securities and Exchange Commission, including its most recent Annual Report on Form 10-K. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200417005086/en/

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Sangamo Appoints D. Mark McClung as Executive Vice President and Chief Business Officer - BioSpace

Money is on the way. At least that's the case if you're among the 80 million Americans who qualify for the coronavirus stimulus checks funded by theCoronavirus Aid, Relief, and. Economic Security (CARES) Act signed into law last month.

For too many, the $1,200 payment will be needed simply to make ends meet. Others, though, will have extra money to spend or to invest. If you're in the latter group, investing your stimulus check makes a lot of sense with the stock market still down by a double-digit percentage from the highs set earlier this year.

But which stocks are great picks that could generate explosive growth over the next several years? Here are three stocks to buy to get a big bang for the buck with your coronavirus stimulus check.

Image source: Getty Images.

Shares of MongoDB (NASDAQ:MDB) sank as much as 45% in mid-March before bouncing back nicely. But the stock is still down well below its previous highs. I think buying MongoDB now will enable patient investors to reap tremendous gains over the long run.

The last two letters of MongoDB's name reveal the company's focus on the database market. But MongoDB's database is different from the big players in the industry in a couple of important ways. First, it was designed from the ground up for the unstructured data that's being generated in massive quantities today, like images and videos. Second, MongoDB created its database to be run from anywhere, including the cloud.

I really like the first part of the company's name, though, because it hints at the size of the opportunity that lies ahead. "Mongo" is short for "humongous." And MongoDB definitely has a humongous opportunity. The global database market is expected to grow to $97 billion by 2023 from $71 billion this year. MongoDB currently captures less than 1% of the market, but it's growing faster than its much larger rivals.

A major key to MongoDB's success is its Atlas cloud-based database-as-a-service. The company reported 80% year-over-year sales growth for Atlas in Q2, with the fully managed cloud database now generating 41% of MongoDB's total revenue. I expect Atlas will continue to fuel MongoDB's tremendous growth and help the company snag a lot more of the expanding global database market over the next five years.

Fastly's(NYSE:FSLY) name also hints at what the company does -- delivering web content to users faster than other technologies. The company's platform moves data and applications closer to users at the edge of the network, the point right before an organization loses control of its data.

You could also say that the company's name describes its own growth. Fastly reported 44% year-over-year revenue growth in the fourth quarter of 2019. Its number of enterprise customers in Q4 increased to 288 with an average spend of $607,000, up from 274 enterprise customers in Q3 with an average spend of $575,000.

The edge cloud platform and content delivery network technology offered by Fastly will almost certainly enjoy even greater demand in the future with the rise of 5G wireless networks. While major cloud services providers including Amazon.comand Microsoftwill compete against Fastly, the growth in the overall market should be enough for multiple winners.

Fastly stock now trades at a double-digit percentage discount from earlier this year. It's still expensive based on conventional valuation metrics but with its strong growth prospects, I think this tech stock will be a winner over the long term.

In keeping with our theme of what's in a name,Editas Medicine (NASDAQ:EDIT)also picked its corporate name to indicate the business it's in. The company is a leader in the development of gene-editing therapies that use CRISPR, a method of gene editing that has been hailed as the biotech discovery of the century.

Unlike MongoDB and Fastly, Editas can't boast of impressive revenue growth yet. That's because the company is still several years away from the possibility of winning regulatory approval for its first drug. But Editas is making progress toward the goal of launching its first product.

In March, Editas and its partner, Allergan, dosed the first patient in the world's first clinical study in humans evaluating an in vivo (inside the body) CRISPR gene-editing therapy. This study will assess the safety, efficacy, and tolerability of a CRISPR therapy targeting Leber congenital amaurosis type 10 (LCA10), the most common cause of genetic childhood blindness.

Editas is also working on advancing a promising CRISPR therapy for treating rare genetic disorder sickle cell disease and hopes to file for approval by the end of 2020 to begin a clinical study. It's also developing an experimental CRISPR therapy for treating solid tumors. The biotech stock is the riskiest of these three, but if Editas is successful with its gene-editing programs it could potentially be the biggest winner of all.

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3 Stocks to Buy to Get a Big Bang for the Buck With Your Coronavirus Stimulus Check - The Motley Fool

Newswise Pancreatic cancer is expected to become the second-deadliest cancer in the United States by 2030, driven in part by rising obesity rates. A new study led by Yale Cancer Center (YCC) researchers has demonstrated in mice that hormones released from the pancreas itself can advance pancreatic cancer and that weight loss can stop this process in its early stages. The research was published today in the journal Cell.

These discoveries raise the hope of finding new ways to prevent both the growth and the spread of pancreatic cancer, said Mandar Muzumdar, M.D., assistant professor of genetics at Yale School of Medicine, a member of the Yale Cancer Biology Institute, and senior author of the paper.

Muzumdar and his collaborators, including a team from the Massachusetts Institute of Technology (MIT), began the project with a mouse model that was genetically modified to develop pre-cancerous pancreatic lesions with a mutation in the KRAS gene, which is mutated in most human pancreatic cancers. The mice were also genetically engineered both to become obese and to rapidly lose weight when scientists administered an additional form of genetic manipulation or limited their food intake.

Unlike mice of normal weight engineered with the KRAS mutation, obese mice with this mutation rapidly developed advanced pancreatic cancer. And unlike most humans diagnosed with the disease, tumors in the model mice did not present additional genetic mutations that would further enable tumor progression. Just by making them obese, we could essentially simulate the effect of an additional mutation, Muzumdar said. That suggested that there is a huge effect of obesity on cancer development in mice.

Next, he and his colleagues studied whether weight loss could slow the development of cancer in the mice who had developed pre-cancerous lesions. The results were dramatic. We found that if we made the mice lose weight prior to advanced cancer development, we could essentially block the progression to advanced cancer almost as if they were never obese, Muzumdar said. If we made the mice lose weight after advanced cancers had developed, the mice still succumbed to the disease within the same timeframe.

The finding suggested the possibility of intercepting tumor formation or progression by weight loss, or eventually using novel drugs that target the underlying biological pathways. Muzumdar noted that the result matches up well with clinical studies of people who are given weight-reducing bariatric surgery, which appears to lower the risk of developing pancreatic cancer.

To determine the cause of the tumors in these mice, the team looked at the genes being expressed in clusters of hormone-producing cells called islets in their pancreases. One type of pancreatic islet cell known as a beta cell normally produces insulin. In these mice, however, some beta cells were churning out a hormone called cholecystokinin (Cck), normally generated in the intestine to aid digestion. These beta cells also seemed to secrete less insulin. This finding interested Muzumdar and his team because they knew that the Cck hormone acts on the digestive enzyme-secreting cells where the predominant type of pancreatic cancer emerges.

They also discovered that Cck expression in the islets dropped when the obese mice lost weight. Additionally, other mouse models that were engineered with the KRAS mutation and forced to express Cck in beta cells, but were not obese, were more likely to form pancreatic tumors than mice engineered with the KRAS mutation alone.

Muzumdars lab is now studying why beta cells switch from making insulin to Cck. Another key puzzle is how Cck can boost tumor formation and progression. Our hope is that the underlying pathways and mechanisms were identifying in obesity also may apply to those who develop pancreatic cancer in the absence of obesity, he added.

Lead authors on the paper include Jaffarguriqbal Singh and Lauren Lawres from Yale and Katherine Minjee Chung and Kimberly Judith Dorans from MITs Koch Institute of Integrative Cancer Research. Yales Cathy Garcia, Daniel Burkhardt, Rebecca Cardone, Xiaojian Zhao, Richard Kibbey, Smita Krishnaswamy and Charles Fuchs contributed to the study, as did Rebecca Robbins, Arjun Bhutkar and Tyler Jacks from MIT. Other contributors included Ana Babic, Sara Vayrynen, Andressa Dias Costa and Brian Wolpin from Dana-Farber Cancer Institute; Jonathan Nowak from Brigham and Womens Hospital; Daniel Chang of Stanford Cancer Institute; Richard Dunne and Aram Hezel of the University of Rochester Medical Center; Albert Koong of the University of Texas MD Anderson Cancer Center; Joshua Wilhelm and Melena Bellin of the University of Minnesota Medical Center; and Vibe Nylander, Anna Gloyn and Mark McCarthy of the University of Oxford.

Funding for the study was provided by the Lustgarten Foundation, the National Institutes of Health, and YCC.

About Yale Cancer Center and Smilow Cancer HospitalYale Cancer Center (YCC) is one of only 51 National Cancer Institute (NCI-designated comprehensive cancer) centers in the nation and the only such center in Connecticut. Cancer treatment for patients is available at Smilow Cancer Hospital through 13 multidisciplinary teams and at 15 Smilow Cancer Hospital Care Centers in Connecticut and Rhode Island. Smilow Cancer Hospital is accredited by the Commission on Cancer, a Quality program of the American College of Surgeons. Comprehensive cancer centers play a vital role in the advancement of the NCIs goal of reducing morbidity and mortality from cancer through scientific research, cancer prevention, and innovative cancer treatment.

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Study points to obesity as driver of pancreatic cancer - Newswise