Genetic study uncovers potential new treatments for inflammatory ... Science Daily Researchers have studied over ten million DNA variations and found new links between the human genome and inflammation tracers. The study uncovered new ... |
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Genetic study uncovers potential new treatments for inflammatory ... - Science Daily
Forbes | How Will Virtual Reality Change The Healthcare Industry? Forbes Virtual Reality has already changed the healthcare industry. Moreover, healthcare is one of the hottest industries, where Virtual Reality is rapidly hitting a place for itself. Let's see a few examples: Relief of the sensation of pain. Here, our ... |
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How Will Virtual Reality Change The Healthcare Industry? - Forbes
In an interview with Fox News conservative commentator Bill O'Reilly asked: "Can Americans in 2017 expect a new health care plan rolled out by the Trump administration this year?"
"Yes, in the process and maybe it'll take till sometime into next year but we're certainly going to be in the process," Trump said.
"(It's) very complicated -- Obamacare is a disaster. You have to remember, Obamacare doesn't work so we are putting in a wonderful plan," he said. "It statutorily takes a while to get. We're going to be putting it in fairly soon, I think that -- yes, I would like to say by the end of the year at least the rudiments but we should have something within the year and the following year."
It's unclear what Trump meant when he said it will take until sometime next year. He had promised to unveil a plan once his Health Secretary nominee, Tom Price, is confirmed. That's expected to happen later this month.
Trump, however, appears to be acknowledging what Republicans in Congress are also discovering -- that it's not so simple to dismantle the sweeping health reform law. Lawmakers are also slowing the time line to repeal and replace major sections of the law. Some are even talking about repairing it first.
Even after Congress passes a replacement plan and Trump signs it, Obamacare will likely still remain in effect for a year or two. It will take time for insurers and agencies to shift to the new Republican plan.
Trump also answered affirmatively when asked if taxes will be slashed this year.
"I think so, yes," Trump said. "And I think that before the end of the year I would like to say yes."
CNN's Tami Luhby contributed to this report.
This story has been updated.
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Crain's Chicago Business | 'Health care deserts' clinic operator inks deal with Medicare insurer Crain's Chicago Business Medicare Advantage insurer MeridianCare has entered into a value-based contract with Oak Street Health, a Chicago-based network of 19 primary-care clinics serving the Medicare population. The partnership will allow MeridianCare patients to seek ... |
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'Health care deserts' clinic operator inks deal with Medicare insurer - Crain's Chicago Business
To the editor: Thanks to David Lazarus for reporting on a bill to replace the Affordable Care Act with a Medicare for all alternative that would bring us up to par with other industrialized nations. (Looking for a really good Obamacare replacement? Here it is, Feb. 3)
There are aspects of this plan that rarely get discussed. Imagine what it would be like for employers to unload the burden of paying for health insurance. Workers compensation policies would no longer be necessary. Medical portions of auto insurance could be eliminated since everyone would already be covered.
Certainly, the insurance industry would not be happy. Yet how much longer must we be held captive to its increasing rates and declining quality of product?
Tom McGee, Oxnard
..
To the editor: Calling for a single-payer healthcare system based on Medicare ignores the fact that Medicare relies on the much larger pool of private healthcare plans to make up for shortfalls in the rates of reimbursement.
Because of low Medicare reimbursement rates, many doctors no longer accept it. We were able to remain with our long time doctor when we transitioned to Medicare only because he grandfathered us in; he does not accept new Medicare patients.
Moving to a healthcare system where the government decides the value of the services will drive doctors to provide a level of care commensurate with the payment received. Will Americans settle for a model that is more heavily weighted to reduce cost than elevate the excellence of care?
And where will we get all the foreign doctors willing to work for Medicare-like levels of payment?
Edward Hull, Seal Beach
..
To the editor: I think we could expand Americans lifespans by simply not stressing out about paying for our healthcare. Having to rely on GoFundMe campaigns to cover the medical costs for ourselves and loved ones is a shameful example of how our healthcare system does not work.
Some might call Medicare for all socialism; I call it compassionate.
Celeste Demetor, Yorba Linda
Follow the Opinion section on Twitter @latimesopinion and Facebook
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The benefits of Medicare for all extend beyond universal healthcare - Los Angeles Times
States Prove Why Direct Primary Care Should Be A Key Component To Any Health Care Reform Plan Forbes Direct primary care (DPC) will end up playing a more prominent role on the national stage of conservative health reform. The Wall Street Journal considers this patient-centered delivery model the fourth leg to any plan that would replace Obamacare. |
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When it comes to designing a future-ready infrastructure, IT leaders need to dream big. If you are an informaticist, IT leader, clinician, or purveyor of IT solutions for the healthcare industry, realizing the future-ready IT environment you want requires imagination and planning.
As the healthcare IT world converges in Orlando for HIMSS17, think of designing your infrastructure of the future in the same way that you map out a trip to a theme park. Zero in on the destinations that will deliver the best overall experience, consult with others who have been there, and go in knowing that sometimes you have to think outside the box.
The road to a healthcare digital workspace includes stops at these critical destinations: security, mobility, automated workflows, and cloud capabilities.
Carefully examining how these roads intersect in your healthcare environment could make the difference between user satisfaction and an environment that doesnt hit the mark.
Allegro Pediatrics provides a case in point for why security matters so much. CIO Brock Morris frames the need for secure healthcare solutions in the context of the multiple challenges that the entire industry faces. Healthcare is highly regulated. New mandates are issued every day. Not only must Patient Health Information (PHI) be transmitted securely, but its access must also be tightly controlled. Security must be an enabler rather than a roadblock to getting work done. This Washington-based pediatric practice met the stringent need for built-in security by ensuring that the technology it chose had security built in across the entire solution stack.
Allegro Pediatrics also understood the tie between security and mobility. It implemented secure mobility strategies that make clinicians more productive anywhere, on any device, and over any network. Allegros IT team replaced workstations in exam rooms with individual laptops and tablets. Ninety physicians charged with seeing 250,000 patients annually gained the ability to access PHI with lightning speed onsite or from the remote workplace of their choice.
On a somewhat larger scale, Saint Marys Health System of Waterbury, Connecticut, wanted to simplify IT management. Ultimately, that translated into improving and automating workflows. It also meant implementing new technology to reduce the number of clicks and reboots for roaming clinicians and building a team comprised of experts in the field of clinical workflow. Informaticists worked in tandem with IT professionals to design innovative technology solutions. Birgit Koellmer, RN, nurse informaticist, and clinical workflow expert, and Tom Calo, solution engineer, actually made rounds in the hospital with doctors and medical professionals. They married technology and the specialty of designing efficient workflows to improve how users accessed information and managed patient visits. The result was truly a culture change that enabled better patient care.
Last, but not least, consider cloud capabilities. Historically, healthcare IT has been slow to adopt cloud infrastructure, but thats changing rapidly. If you havent explored how your organization can implement cloud solution, now is the time to think outside the box. Ideally, a cloud solution for healthcare addresses many of the same concerns that it does in other industries: technology migration and continual upgrades, IT resource constraints, mobility and business continuity. All of this should be shored up by a foundation of security.
Chart your destination carefully. Ensure that security, mobility, automated workflows and cloud capabilities all intersect in the right places. Youll get there!
To join the ongoing dialogue about digital workspaces, follow us on Twitter @CitrixHealth, and visit us at HIMSS17 (Booth #2623).
About the Author:Kathy Holoman, Portfolio Marketing Healthcare, Citrix
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4 must-see destinations on the way to healthcare's digital workspace dream - Healthcare IT News
PHOENIX - When a child is diagnosed with cancer, it can be emotionally overwhelming. At Phoenix Childrens, more than 300 children were diagnosed with pediatric cancer last year. The good news is, with 50 percent of these children involved in clinical trials, cure rates have jumped to 90 percent for most types of pediatric leukemia.
Jessica Boklan, MD is the director of clinical oncology research and the Early Drug Development Program, and co-director of the Leukemia Program at Phoenix Children's. She supervises all oncology clinical trials carried out at the hospital, concentrating on evaluating new drugs for patients who do not respond to currently available pediatric cancer treatments.
A new and innovative treatment, known as precision medicine, has surfaced within the past few years. Precision, or personalized, medicine uses genetic information to determine the right treatment for the right patient at the right time. By studying a patient's genetic makeup, researchers can identify their susceptibility to disease, predict their response to a particular drug and match the patient with a personalized therapy.
In May 2016, Hannah and Brooklyn were diagnosed with acute lymphoblastic leukemia, cancer of the blood. The girls received their diagnosis one day apart from each other. Under the care of Dr. Boklan, they receive treatment one to two times each week at the Center for Cancer and Blood Disorders at Phoenix Childrens. Since their diagnosis, Hannah and Brooklyn, as well as their families, have become the best of friends.
The Center for Cancer and Blood Disorders at Phoenix Children's Hospital is the largest pediatric program of its kind in Arizona, providing complete care for children diagnosed with malignancies and hematologic diseases. Call (602) 933-0920 for information.
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Precision-medicine approach could revive prostate cancer test Science Daily A new study led by researchers at UC San Francisco and Kaiser Permanente has identified genetic predictors of normal prostate-specific antigen (PSA) levels in healthy men, which could be used to improve the accuracy of PSA-based prostate cancer ... |
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Precision-medicine approach could revive prostate cancer test - Science Daily
New Vision | Research consortium identifies 13 new genetic regions associated with COPD and shared risk factors for pulmonary ... Science Daily ... only uncovered new genetic risk factors for COPD, but also shown overlap of COPD genetic risk with the risk to asthma and pulmonary fibrosis," said lead author Brian Hobbs, MD, MMSc a physician-researcher in the Channing Division of Network ... World lung health study allows scientists to predict your chance of developing COPD |
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Paris (AFP) - Gene therapy delivered by a benign virus enabled deaf lab mice to hear for the first time, researchers said Monday, offering hope for people with genetic hearing impairments.
The breakthrough could pave the way for gene-based treatments, they reported in two studies, published in Nature Biotechnology.
"With more than 100 genes already known to cause deafness in humans, there are many patients who may eventually benefit from this technology," said Konstantina Stankovic, a professor at Harvard Medical School.
Genetic hearing disorders affect some 125 million people worldwide, according to the World Health Organization.
An expert not involved in the research welcomed the findings as "very encouraging", but cautioned the technique has yet to be proven safe, and that human trials are likely years away.
In the first study, Stankovic and colleagues used a harmless virus to transport -- deep into the mouse ear -- a gene that can fix a specific form of hereditary deafness.
Previous attempts had failed, but this time the viral package was delivered to the right address: the so-called outer hair cells that "tune" the inner ear to sound waves.
"Outer hair cells amplify sound, allowing inner hair cells to send a stronger signal to the brain," explained Gwenaelle Geleoc, a researcher at the F.M. Kirby Neurobiology Center at Boston Children's Hospital.
The technique bestowed hearing and balance "to a level that's never been achieved before," she said in a statement.
"Now you can whisper, and the mice can hear you."
In the second study, a team led by Geleoc used the same viral courier to treat mice with a mutated gene responsible for Usher syndrome, a rare childhood genetic disease that causes deafness, loss of balance, and in some cases blindness.
The virus carried a normal version of the same gene to damaged ear hair cells soon after the mice were born.
- Narrow time window -
The results far exceeded anything to date: 19 of 25 treated mice heard sounds quieter than 80 decibels. Normal human conversation is about 70 decibels.
A few of the mice could hear sounds as soft as 25 to 30 decibels -- roughly equivalent to whispering.
According to Margaret Kenna, a specialist in genetic hearing loss at Boston Children's Hospital not involved in the studies, "cochlear implants are great, but your own hearing is better."
Electronic implants work by bypassing damaged hair cells in the ear to send sound signals directly to the brain.
"Anything that could stabilise or improve native hearing at an early age would give a huge boost to a child's ability to learn and use spoken language," she said.
The need for early intervention, however, could be a problem in itself, other experts pointed out.
In humans, such an intervention would ideally have to happen before a child is born, said Jonathan Ashmore, a professor at University College London's Ear Institute.
Alan Boyd, president of Britain's Faculty of Pharmaceutical Medicine hailed "a very encouraging result".
"But it is only a mouse model," he cautioned, noting that it is still unknown how the human immune system might react.
Any gene deafness treatment is "at least three years away, if not more," Boyd conjectured.
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A large-scale international study of more than 700,000 adults reveals 83 new genetic variations that control human height. Understanding the genetics of human height may help to develop genetic tools to predict a person's risk for common diseases, suggest researchers.
The Genetic Investigation of Anthropometric Traits (GIANT) Consortium - an international collaboration that researches the genetics that modulate human body size and shape, including measures of height and obesity - discovered the new genetic variations.
Previous studies have used genome-wide association studies (GWAS) to locate genetic variants. This method rapidly scans across the genomes of large populations for markers that track with a particular trait.
GWAS is successful at finding common genetic variants. However, most of these only alter height by under 1 millimeter. GWAS unsuccessfully captures uncommon genetic variants that may have a larger affect on height.
Another problem is that common genetic variants that track with traits lie outside the protein-coding parts of genes. This positioning makes it harder to find out which genes they affect.
In the new study, the investigators decided to use another technology called the ExomeChip in order to overcome some of the issues experienced with GWAS. ExomeChip tested for almost 200,000 known genetic variants that are less common in the 711,428 adults included in the study.
These known variants can be used as a shortcut to work out the genes that are important for particular diseases or traits. Most of these variants had not yet been assessed in previous studies of height.
Out of the 83 uncommon variants identified that affect human height, 51 of them were "low-frequency" variants that are found in less than 5 percent of people, and 32 of them were rare variants found in less than 0.5 percent.
"Of these 83 genetic variations, some influence adult height by more than 2 centimeters, which is enormous," says Guillaume Lettre, a professor at the Universit de Montral's Faculty of Medicine and researcher at the Montreal Heart Institute. "The genes affected by these genetic variations modulate, among other things, bone and cartilage development and growth hormone production and activation," he adds.
Lettre co-led the study with professors Joel Hirschhorn - of Boston's Children's Hospital, MA, and the Broad Institute of MIT and Harvard, and chair of the GIANT Consortium - and Panos Deloukas - of the Queen Mary University of London in the United Kingdom. Almost 280 other research groups were also involved.
The team notes that 27.4 percent of the heritability of height is now accounted for, with most heritability still explained by common genetic variants.
The study also found several genes that could potentially be targeted therapeutically for children with growth problems.
One of the genes, STC2, had DNA changes that significantly affected height. Genetic changes that inactivate the STC2 gene increase the height of individuals who carry them in their DNA by 1-2 cm, by acting on certain growth factors. "In this sense, evaluating whether drugs that block STC2 activity could have an impact on growth seems to us very promising," says Lettre.
"The success of our study was due to our large sample size," says Prof. Deloukas. "Our results suggest that our genetic approach works. We can now start identifying similar genetic variations that may influence the risk of developing common diseases such as diabetes, cancer, schizophrenia, and cardiovascular disease, to name just a few."
Lettre says that adult height was used as a simple trait to understand how information in DNA can explain how individuals are different. "The idea was that if we could understand the genetics of human height, we could then apply this knowledge to develop genetic tools to predict other traits or the risk of developing common diseases," he adds.
Precision medicine is an emerging approach that involves customizing treatments and prevention measures to an individual patient. Lettre and colleagues suggest that the findings of the study could help to identify genetic variations that increase a person's risk of developing diseases. If this were the case, pinpointing these variations would be valuable in precision medicine.
"This study has shown that rare protein-altering variants can be helpful at finding some of the important genes, but also that even larger sample sizes will be needed to completely understand the genetic and biologic basis of human growth and other multifactorial diseases."
Prof. Hirschhorn
The GIANT Consortium's future work will focus on a GWAS of height, including more than 2 million people and studies involving sequencing data. "We predict that these more comprehensive studies will continue to enhance our understanding of human growth and how best to attain the biological insights that will inform treatments for common diseases," concludes Hirschhorn.
Learn about a study that has uncovered 14 new genetic disorders in children.
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DNA changes that affect human height uncovered - Medical News Today
Editors note: This piece is written byDr. Miller, a physician and molecular biologist, who was the founding director of the FDAs Office of Biotechnology.
Genetic engineers have developed a way to produce the two principal components [of blue jeans], cotton fabric and indigo dye, for less money and soon will make commercial blue jean production cheaper than ever.
Bt cotton helps farmers to control major peststhe cotton and pink bollworm and the tobacco budwormwhich account for a quarter of all crop destruction due to insects. From 1996 through 2014, this technology increased cotton yields by an average of 17.3%
Bt cotton is also environmentally friendly. With conventional cotton, farmers control insects by applying huge amounts of chemical pesticides known to harm birds, fish and other aquatic organisms. Lessening the need for pesticides also reduces farm workers exposure to those chemicals.
The other main ingredient in bluejeans, indigo dye, is usually produced synthetically through a complex, multistep process performed with highly toxic chemicals. It requires special facilities and precautions to protect workers and the environment. But indigo dye can also be made using genetically engineered bacteria. This process has fewer steps, uses water instead of toxic organic solvents, incorporates corn syrup as the primary starting material, and yields nontoxic waste products. While it is not yet efficient enough for commercial use, stay tuned.
Instead of accepting critics unsubstantiated claims, consumers should be demanding [genetic engineerings] wider application in agriculture and other industries.
The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Youd Look Good in Designer Genes
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Scientists have used a gene-editing technology to make cows more resistant to tuberculosis.
The researchers used a tool called CRISPR-Cas9, by which scientists can make changes to DNA in order to potentially make creatures more resistant to diseases, correct detrimental genetic mutations and other applications. In this case, they inserted a gene into cows that would make them resistant to bovine tuberculosis, then successfully bred that resistance into their offspring. Their findings were reported in the journal Genome Biology, with the authors saying the result demonstrates a possible use of the technology and contributes to the concept of gene-editing for agricultural purposes.
Read: The Danger of a Genetically Engineered Virus
Importantly, our method produced no off-target effects on the cow genetics, lead author Dr. Yong Zhang explained, according to the Daily Mail.
A gene-editing technology has created cows that are resistant to tuberculosis. Pixabay. public domain
The researchers from Northwest A&F University in Xianyang, China, wrote in their study that those off-target effects unintended and unrelated results are an issue when it comes to animals whose genes have been purposely modified.
Although it holds great potential to cure or treat disease and other ailments, CRISPR remains a controversial technology, with some people fearing it will be used to create designer babies or be used for unethical purposes.
Source: Zhang Y, Gao Y, Wu H, et al. Single Cas9 nickase induced generation of NRAMP1 knockin cattle with reduced off-target effects. Genome Biology. 2017.
See also:
How Milk Is Made and Why Humans Drink It
6 Signs of Lactose Intolerance
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Genetic Engineering: CRISPR Technology Makes Cows Resist ... - Medical Daily
Several years ago, in its Retro Report section, the New York Times posted an old video about the genetically modified Flavr Savr tomato, which was developed by Calgene and launched on to the market in 1994 only to be withdrawn a few years later. The video includes clips of a television program from the time. An intrigued woman is shown two tomatoes picked 30 days earlier, neither of which has been refrigerated. The first tomato is perfect: round, bright red and with no signs of softening. The second has wrinkly skin and a dulled color, clearly rotten. The perfect tomato is a Flavr Savr, engineered to maintain the texture, juiciness and color of a freshly picked tomato for longer. However, despite its apparent perfection and characteristics which, from a commercial point of view, should have certainly made it a success, the Flavr Savr vanished not long after it appeared. Why? Because it was missing the one feature more important than any other: flavor.
Fast forward to today, and the latest cover of Science magazine features Tastier Tomatoes, which hints at the research being conducted by a large group of scientists to design a truly perfect tomato with the texture, juiciness and color of a freshly picked tomato, and indeed, the flavor of heirloom tomato varieties.
The premise of the study is that modern commercial tomato varieties are substantially less flavorful than heirloom varieties. Over time, agricultural research has focused on improving the characteristics that determine whether different varieties are commercially successful: yield, disease resistance and firmness. All at the expense of flavor. Often, the tomatoes we buy taste of nothing. They seem like fake fruit, all too perfect to look at, but flavorless. To fix this fault, the team of scientists have studied the characteristics that most affect the flavor of the product, sequencing the whole genome of 398 modern, heirloom and wild varieties. They then selected 160 tomato samples from 100 varieties and grew them in the laboratory, harvested them and submitted them to extensive taste testing by 100 people. The participants voted for the tomatoes based on flavor and, by comparing this information with their genetic analyses, the scientists determined which genes were associated with flavors that the public enjoyed.
Is a new future taking shape for a fruit that the FAO considers to be one of the most high-value in the world? Maybe. A laboratory-made future, completely removed from the land and restricted by private patents, like all genetically modified products. Slow Food on the other hand, supports a different kind of research, namely what farmers have been doing for around 10,000 years: selecting seeds, conserving them, propagating them and developing varieties suited to different soils and climates, based on traditional knowledge. Work that, over centuries, improves the yield, flavor and nutritional value of crops, without compromising biodiversity and, on the contrary, gradually enriching it.
Examples of these crops are cataloged in the Ark of Taste and among Slow Food Presidia: the Platense tomato from Argentina which, despite its far superior flavor compared with commercial tomatoes, has to deal with intense competition from high-yield hybrid varieties that can be produced all year round; the Smooth Skin Geraldton tomato from Australia, which is suffering due to the appearance of greenhouses in Melbourne and Adelaide which enable tomato production all year round; Kurtovo Konare pink tomatoes, whose survival is under threat from foreign varieties with higher yields that are more suited to being transported; and the Torre Canne Regina tomato, grown without irrigation using organic methods in Apulia, which faces almost unbeatable commercial competition from greenhouse-grown cherry tomatoes. We could mention dozens of other such examples of tomatoes that farmers have developed over centuries through careful selection, rather than artificially engineered in the laboratory. And we would prefer a future where the value of naturally flavorful tomatoes is appreciated once more.
Images: Science Magazine, Western Gardens
First offical Slow Food conference in Iran
Slow Food rememebers Predrag Matvejevic
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Saving the flavors of centuries: against Flavr Savr and the genetic engineering of taste - Slow food
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February is National Cancer Prevention Month. It's a disease that more than a million Americans are diagnosed with each year, according to the National Cancer Institute. This morning in our special series "War on Cancer," TODAY takes a look at the latest advances in the fight against this deadly disease.
Celine Ryan, a 51-year-old engineer and mother of five, was diagnosed with stage 4 colon cancer three years ago. After undergoing surgery, radiation and chemotherapy, doctors discovered cancer in her lungs seven tumors that threatened her life.
Ryan, who lives in Michigan, read about a clinical trial using gene therapy at the National Cancer Institute in Bethesda, Maryland, and decided to apply. The trial is headed up by Dr. Steven Rosenberg, a leading researcher in immunotherapy at the institute. She decided the treatment would be a birthday present to herself.
Getting into the trial wasn't simple because her tumors, though numerous, weren't large enough for the form of treatment being tested, but she was finally accepted in March 2015.
RELATED: Keytruda, the drug that helped Jimmy Carter, also can stop lung cancer
The treatment involves removing cells from a tumor your body's own cancer-fighting cells multiplying them by billions in a lab, then returning them back to your body to fight the tumor.
Celine Ryan is being referred to as an historic figure in medicine.
After spending a month in the hospital and letting the treatment run its course, six of her seven tumors had completely disappeared. The last tumor started to grow eight or nine months later and the decision was to remove it through surgery.
RELATED: 10 things I wish I knew before I was diagnosed with breast cancer
The treatment isn't widely available now, and not all patients experience the positive results Ryan had.
"Many have not responded," Rosenberg said. "But from every patient that we treat, whether... their cancers go away or not, we learn something."
Today, Celine Ryan is ten months cancer-free.
Thanks to Ryan's unusual genetic makeup, researchers were able to identify how to attack the mutation that causes common cancers. This experimental treatment may not be the solution for everyone, but for Ryan, it's meant ten months of being cancer-free.
"We can do, and are planning to do, that kind of gene therapy using the exact receptor we got from Celine's cells to treat other people," Rosenberg explained.
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The experimental gene therapy treatment that helped one woman fight cancer - Today.com
Scientists have restored hearing in deaf mice down to a whisper using an improved gene therapy in what is being described as a landmark study with unprecedented results.
A research team from the Boston Childrens Hospital initially conducted a study with Harvard Medical School in 2015 that restored rudimentary hearing in genetically deaf mice using gene therapy.
In this new study, however, the Boston team managed to restore hearing in deaf mice down to 25 decibels - or the equivalent of a whisper - using an advanced gene therapy developed at Massachusetts Eye and Ear.
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The team said they sought to advance gene therapy to treat genetic deafness as there is currently no biological treatments for hearing loss.
We focused on Usher syndrome, a devastating genetic disorder that causes blindness, balance disorders and profound deafness,read the study, published in Nature Biotechnology Monday.
Using a synthetic adeno-associated viral vector, the team transduced 80-90 percent of sensory hair cells, resulting in a recovery of gene and protein expression, restoration of sensory cell function, rescue of complex auditory function and recovery of hearing and balance behavior to near wild-type levels.
The end result of the study saw an unprecedented recovery of inner ear function that suggests the biological therapies may be suitable to treat deafness in humans with genetic inner ear disorders.
"With more than 100 genes already known to cause deafness in humans, there are many patients who may eventually benefit from this technology,"said Dr. Konstantina Stankovic, a senior investigator in the first study with Harvard Medical School.
READ MORE: Rat-mouse interspecies transplant brings hope human organs could be grown in animals
The next step toward treating human patients is to test the gene therapy in larger animals.
"This is a landmark study,"says Dr. Jeffrey R. Holt, director of otolaryngology research at Boston Children's Hospital, and co-author on the paper.
"Here we show, for the first time, that by delivering the correct gene sequence to a large number of sensory cells in the ear, we can restore both hearing and balance to near-normal levels."
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Scientists restore hearing in deaf mice using advanced gene therapy - RT
The hottest biotechs in the field of hemophilia are stealing the show at this years edition of the EAHAD hemophilia congress in Paris.
A disorder for which no cure is available, hemophilia is caused by absent or defective genes coding for blood clotting factors, turning simple injuries intohealth risks and causing spontaneous bleeding. Researchers and companies worldwide working to improve hemophilia therapy are meeting this week in Paris for the10th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).
We recently reviewed the latest advances in hemophilia, a field teeming with innovative solutions and technology.Among the most interesting presented at the congress are new results on gene therapy and RNAi, a pen to treat hemophilia and many companies fighting to reduce thedosing frequency of prophylactic therapy. On top of that, Shire has reported that current estimates of people suffering from the disease could be completely wrong
Novo Nordisk ismaking plans to use its famous insulin pens to deliver hemophilia drugs. According to astudy evaluating user experience with the pens presented at the congress, participants liked the device as it is easy to use, well designed, more portable and involving fewer steps than their current kits for hemophilia.
The ultimate goal of the Danish company is to use its FlexTouch pen to deliverconcizumab, an antibody againsttissue factor pathway inhibitor (TFPI) currently in Phase I for both hemophilia A and B.
Shire has presented a study revealing that the incidence of hemophilia could be more than three times higherthan current estimates. It also showed that only 25% of hemophiliacs receive adequate treatment. These findings might push efforts to put an end to this situation and stimulate market growth.
Shirepresented positive results from a Phase II/III trial for Adynovate (BAX 855) in children with hemophilia A. Interestingly, the company also showed early stage in vitro results for combination therapies with a biosimilar of Roches emicizumab (ACE910). It looks like the antibody, not yet in the market, already has strong competitors getting ready for when its patent expires.
Sobi, inSweden, has co-developed recombinant clotting factors with an extended half-life in partnership with Biogens spin-offBioverativ. To do so, they fuse the clotting factor to the Fc portion immunoglobulin G1 proteins.
The team has presented positive long-term safety and efficacy results forEloctatein hemophilia A andAlprolixin hemophilia B. Both are already in the market and reduce dosing frequency to weekly injections.
OPKO Biologics, in Israel, follows a strategy similar to Sobis. ItsCTP technologyextends the half-life of proteins by fusing them with theC-terminal peptide of human chorionic gonadotropin (hCG).
The company has presented data forMOD-5014(FVIIa-CTP) supporting the advance intoPhase II/IIItrials. The drug is intended for delivery twice a week, which is double of that from Sobis products.
Spark Therapeuticsis one of the leaders in the development ofgene therapy for hemophiliaanduniQures main competitor. The American company will report results from itsPhase I/IItrial forSPK-9001in hemophilia B showing sustained activity of the therapy after12 weeks, with only one reported bleeding.
Despite good results, Spark is facing strong competition from the DutchuniQure. Its gene therapyAMT-060has already shown sustained effects for at least52 weeksin a patient subpopulation. Both companies nowhavebreakthrough designation from the FDA and therace to reach the market is tight.
Sanofis partner, Alnylam, is conducting clinical trials across the UK, Switzerland and Bulgaria to test its unique RNAi technology for hemophilia. Its candidate fitusiran, which blocks antithrombin to improve clotting,is proving safe and effective inPhase I/IItrials.
This unique treatment has the potential to reduce dosing to a monthly basis and is suitable for patients with both hemophilia A and B, also including those that have developed resistanceto standard treatments.
Among the companies presenting are many others includingGenentech, Bayer and Catalyst Biosciences. The sheer number of innovative approaches under development is great news. Such a wide arrange of solutions could provide a better quality of life for hemophilia patients, each treatment suited for the particular needs of differentpatients. Especially now that, thanks to Shire, scientists know the number of patients suffering from the condition could actually be much higher.
Images from Sashkin, nobeastsofierce, Roberta Canu, Mond Duang,Art tools, LeonP,Pakpoom Nunjui /Shutterstock.com
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Gene Therapy, RNA and Pens at European Hemophilia Congress in ... - Labiotech.eu (blog)
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Despite the early and in some cases stunning results produced by gene therapy treatments in handfuls of hemophilia patients, significant questions remain about their durability, safety, and how broadly theyll be used if they are ultimately shown to work. The first human data produced by Dimension Therapeutics, one of several companies developing hemophilia gene therapies, are the latest example.
Shares of Cambridge, MA-based Dimension (NASDAQ: DMTX) tumbled more than 49 percent on Tuesday on early data from a Phase 1/2 trial of DTX101, its experimental gene therapy for hemophilia B.
DTX101 boosted the levels of the blood-clotting protein Factor IX in six patients. Those on the higher of two tested doses havent needed other drugs since getting treatment. But five of the six patientsand all three on the higher of the two tested dosesalso saw a rise in liver enzyme levels, indicating an immune reaction to the gene therapy. While none of the five patients have had any safety problems, the liver enzyme spikes have caused a delay for Dimension. The company wont test an even higher dose of DTX101 in patients until it gets feedback from the FDA.
Gene therapy offers the potential of a long-lasting, if not permanent treatment for hemophilia patients, whodepending on how severe their disease ismay need frequent infusions of preventative drugs to stave off dangerous bleeds. A group of experimental gene therapies have been creeping their way forward in clinical trials, accumulating data in dribs and drabs. Spark Therapeutics (NASDAQ: ONCE) and UniQure (NASDAQ: QURE) are the furthest along in hemophilia B, while BioMarin Pharmaceutical (NASDAQ: BMRN) leads the way in the more common hemophilia A.
Each experimental therapy has shown promise helping patients produce meaningful levels of the clotting proteins Factor IX and Factor VIII, respectivelymore than 5 percent of the levels found in normal patients, which many view as the minimum bar for successover the course of a year or more. And Spark and BioMarin have seen much higher numbers than that, in some cases. But there are caveats: Those results have come in small sample sizes, and they have varied patient to patient. Data today from Dimension show the three patients on a low dose of DTX101 had roughly 3 to 4 percent of normal Factor IX levels a year after treatment. The results are earlier for those on a higher dose: 5 and 8 percent, respectively, for two patients 12 weeks post-treatment; 7 percent for a third patient 7 weeks after DTX101.
Additionally, so far, liver enzyme increases have been seen in clinical tests for each of the hemophilia gene therapies. Such increases could indicate that patients immune systems were attacking their liver cells, which are the ones that take up the therapeutic gene and churn out the new clotting protein. Theyre typically treated with a short course of immunosuppressive steroids and havent caused bad side effects so far. But in some cases theyve stifled a response to gene therapy, which is important because it means that certain gene therapies may not workor at least wont work as well as they couldfor some patients who develop neutralizing antibodies. It also means that patients who develop those antibodies wont be eligible for a second dose if the gene therapy wears off. This phenomenon reduces the potential market for the firms developing hemophilia gene therapies. Such immune responses were the impetus behind a deal Spark cut last year with Selecta Biosciences (NASDAQ: SELB), for example.
We continue to explore the therapeutic window for DTX101 as our data mature and in light of the [liver enzyme] rises that appear to be associated with a decline in [Factor IX] activity, CEO Annalisa Jenkins said in a statement.
Heres more on Dimension, and the technical differences between each of the companies developing gene therapies for hemophilia.
Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com
Originally posted here:
Investors Sour on Data Debut For Dimension's Hemophilia Gene Therapy - Xconomy
On this weeks podcast, Drew Ogryzek talks about hiring incentives at his company. Alex Moxin put her node reading on hold, and with help from AdapTech, is solutions building an Event Store (stores events for CQRS solutions) using node.js, which so far includes creating a basic webserver. Shes installed and is learning to use Vimium so that she can use keyboard shortcuts to navigate, and is learning about http (hyper text transfer protocol) response headers.
Meetups around town Alex and Drew attended were Hackernest (hosted by Drew), TechVancouver, and DDD/CQRS/ES hosted by AdapTech.
This week, our featured guest is Nikolas Badminton! Badminton is a researcher and futurist speaker who splits his time between Canada (Vancouver, Toronto and Montreal), USA, and the UK. He provides insights into how people, communities, cities, businesses, and countries are changing with applied exponential technology. Niks primary interests in technology are in mixed reality, Internet of Things, smart cities, artificial intelligence, and renewable energy.
He studied applied psychology and computing in the UK, and specialized in artificial intelligence and linguistics along with social network theory and human-computer interaction. For over 20 years, hes been hacking his way through tech jobs in big data, analytics, advertising, and the sharing economy.
He recently interviewed Edward Snowden at the University of Waterloo, spoke to 1,500 leaders at the Premiers Forum for Natural Resources in Prince George, and you can see him at these upcoming events: Canada Futurists in Vancouver and Toronto; Our Futures Conference at Quest University in Squamish; and the 18th Annual Privacy & Security Conference. He will be leading a panel about mixed reality with innovators in that field.
You can see some of Niks featured work and speaking engagements at NikolasBadminton.com and be sure to check out his Modern Futures Podcast, which will soon rebrand to Exponential Minds. Heard here first on the Vancouver Tech Podcast, Nik will soon be launching Exponential Minds, which will be a content and event network and a worldwide superinfluencers network. He is also launching the Futurists Speakers Agency this month, so do check on http://www.futuristspeakersagency.com soon.
Welcome to the future!
If youre interested in contacting Nik you can reach him at nik@nikbadminton.com or on twitter @NikolasFuturist.
Theme music by A Shell In The Pit from the game Parkitect
The Vancouver Tech Podcast is a weekly show focusing on the growing tech industry in the city of Vancouver. Get caught up on the events and meetups around town, startups, new businesses, developers, designers, community programs, and news. Each episode includes an interview with an outstanding member of our community.
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Vancouver Tech Podcast Ep.62: Nikolas Badminton, futurist - BetaKit