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Harvard and MIT Scientists Win Gene-Editing Patent Fight New York Times The Broad Institute in Cambridge, Mass., will retain potentially lucrative rights to a powerful gene-editing technique that could lead to major advances in medicine and agriculture, the federal Patent and Trademark Office ruled on Wednesday. The ... There's a fine red line between cures, enhancements using gene editing techBioWorld Online What the CRISPR Patent Decision Means for Gene EditingThe Atlantic A Patent Decision on Crispr Gene Editing Favors MITWIRED Phys.Org -NPR -Yahoo News all 116 news articles »

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Harvard and MIT Scientists Win Gene-Editing Patent Fight - New York Times

We could be about to see a golden age of health (Picture: Getty)

Gene editing techniques developed in the last five years could usher in a golden age of health with an effective end to the scourge of cancer and inherited disease being banished forever, a leading British expert predicts.

Dr Edze Westra believes the ability to splice selected DNA into cells with great precision is destined to become super important in the next two decades.

He foresees it transforming the human race by creating generations of people for whom cancer, failing vision, and the diseases of old age or bad genes are distant memories.

The bioscientist from the University of Exeter said: There is always a risk with this kind of technology and fears about designer babies and we have started having discussions about that so we can understand the consequences and long-term risks.

I think in the coming decades gene editing will become super important, and I think we will see it being used to cure all inherited diseases, to cure cancers, to restore sight to people by transplanting genes. I think it will definitely have massive importance.

On Tuesday, two highly influential academic bodies in the US shook up the scientific world with a report that, for the first time, acknowledged the medical potential of editing inherited genes.

The National Academy of Sciences and National Academy of Medicine ruled that gene editing of the human germline eggs, sperm and embryos should not be seen as a red line in medical research.

Many critics insist that powerful new gene editing techniques should never be used to alter inherited DNA.

They argue that such a move would be the start of a slippery slope leading to designer babies with selected features such as blue eyes, high intelligence or sporting prowess.

But the two pillars of the American scientific establishment said that with necessary safeguards, future use of germline gene editing to treat or prevent disease and disability was a realistic possibility that deserves serious consideration.

Dr Westra is taking part in a discussion on gene editing and its potential implications for society at the American Association for the Advancement of Science (AAAS) annual meeting in Boston, Massachusetts.

He said gene editing technology not only held out the promise of fixing genetic faults, but could be used to turn cells into miniature factories that churned out therapeutic chemicals or antibodies.

One application was the use of gene drives that increase the prevalence of a certain trait in a population.

For instance, gene editing machinery placed inside the cells of large numbers of malaria transmitting mosquitoes could prevent them spreading the organism that causes the disease to humans.

It could be a fantastic strategy to deal with some of the worlds biggest problems, said Dr Westra.

In terms of ethics we need to work out what happens if a genetically engineered insect flies out of the window of the lab. Trials into gene drives are already happening in labs for malaria.

The most promising form of gene editing, known as CRISPR/Cas9, was first demonstrated in 2012.

It employs a defence system bacteria use to protect themselves against viruses.

A carefully targeted enzyme is used as chemical scissors that cut through specific sections of double stranded DNA. Then the cells own DNA repair machinery can be exploited to insert the pasted genetic material.

Dr Westra said: Gene editing .. is causing a true revolution in science and medicine, because it allows for very precise DNA surgery.

A mutation in a gene that causes disease can now be repaired using CRISPR.

Read the original here:
Cancer could be wiped off the face of the earth with new gene editing techniques - Metro

February 16, 2017 by Merlin Crossley, The Conversation Gene therapy is growing in its capabilities, but there should be limits to its use. Credit: Shutterstock

If you recognise the words "CRISPR-mediated gene editing", then you'll know that our ability to alter DNA has recently become much more efficient, faster and cheaper.

This has inevitably led to serious discussions about gene therapy, which is the direct modification of someone's DNA to rectify a genetic disorder, such as sickle cell anaemia or haemophilia. And you may also have heard of deliberate genetic enhancement, to realise a healthy person's dreams of improving their genome.

Both of these issues have now been tackled in a comprehensive report on gene editing released today by the US National Academy of Science and National Academy of Medicine.

The message is fairly simple: relax, we've seen this all before, little if any harm has eventuated, and society is well placed to move forward together on this.

A definite maybe

Of all human technologies, recombinant DNA has arguably been one of the safest. There have been multiple benefits in both medicine and agriculture. And the legitimate concerns that arose when viruses were first mixed with bacterial genes, when cloning was first introduced, and when stem cells were developed, have not come to pass.

I cannot list all the benefits here, but if you have received the Hepatitis B vaccine or Australian Ian Fraser's Gardasil vaccine, which protects against cervical cancer viruses, you have been protected from disease thanks to recombinant DNA technology.

However, you probably haven't received somatic gene therapy, which is gene alteration directed at fixing one cell type, such as defective blood or liver cells. This is because this therapy only touches a tiny number of people, probably fewer than 1,000 worldwide, and again the benefits have outweighed the risks.

But there is one new message in the report that will grab the headlines.

That is the view on human germline gene therapy, which entails modifications that would be passed on to children and then to their children. This kind of gene therapy has been considered highly controversial. But this time, instead of a simple no thanks there's a definite maybe, provided the therapy is targeted at a severe disease as a last resort.

There will be alarm in some circles at the very mention of germline gene therapy, although perhaps not from the very few people who might be contemplating such treatment for the sake of their future children.

The authors of the report, who are among the mostly highly respected experts in the world, are well aware that many people will not be comfortable with the thought of germline gene therapy. They stress the need for extensive consultation, the meeting of strict criteria, and close regulation.

But in weighing up safety and efficacy, social and individual benefit, they clearly don't want to see a reflex ban put in place that may limit options if this technology can be used to make the life of some individuals better.

On one hand, they are right. This technology is not a threat to the fabric of society. Nor, I'd say, is this a genie that could not be put back in the bottle; gene editing could be reversed.

Nor, like the Sorcerer's Apprentice's broomsticks, will it multiply and spread when we try to restrain it. This is not like letting slip a virus, cane toads, oozing radioactive waste or carbon emissions into the atmosphere.

Seeking germline gene therapy in order to have a disease-free child would be a choice made at a personal level and those not wishing to participate should never feel compelled to do so.

Except, of course, the children who would not have a say in it. But also for them the risks might well outweigh the benefits. And, one way or another, parents already make life-determining choices for their children and sometimes for their children's children.

Even those seeking germline therapy for the sake of their children would mostly have alternatives, such as preimplantation diagnosis, which itself also has ethical considerations. There are no easy answers here.

So I can understand the report's conclusion, although I also believe there are risks, which I'll mention below.

Hard to abuse

There are other aspects of the report worth mentioning. It confirms that we already do properly regulate laboratory-based gene modifications, and we have learned so much from previous somatic gene therapy efforts that we are well placed to push on safely with both research and somatic treatments. I agree with this.

It also says that actual genetic enhancements should be avoided. There is evidence that society is uncomfortable with the idea of individuals, who are not suffering from disease, improving either themselves through somatic therapy or their bloodlines through germline genetic enhancement.

Some people might want more copies of the p53 tumour suppressing gene or to lose their CCR5 gene, which helps HIV invade cells, in order to give their children possible protection from cancer or HIV respectively, but I'd have to say it isn't worth the risk.

I would add that, ethical reservations aside, the sheer complexity of our genomes, and the rather involved and lengthy process of human reproduction, means that I have no concerns that even the craziest world leader could ever generate an army of super-mutants. Such an ambition would be defeated by not knowing which genes to alter, not to mention the requirement to assemble tens of thousands of surrogate mothers, then wait 20 years for the army to mature.

Yes, it is possible that someone somewhere will attempt germline gene enhancement as a stunt. That would be wrong and dangerous, and a risk for the child. But it would not threaten society any more deeply than many other obscene and regrettable individual crimes that sadly occur every day.

Germline gene therapy is illegal in many countries, and although there is a risk that unfortunate "medical tourism" may occur at some stage, I don't expect this to be a greater problem than the already widespread snake-oil selling that is a feature of many economies.

No emergency

So am I comfortable with this report and confident that it covers the ethical issues? I think it is superbly written. It is accurate, up to date, balanced, thoughtful, and covers experiments, somatic therapy, germline therapy, genetic enhancement, societal responses, and the need for public consultation and careful regulation. There is no emergency here.

My main concern is that raising the prospect of germline gene therapy will trigger discussions that will divert us from more pressing issues.

I do worry that introducing this apex concept as a possibility may increase the number of people who fixate on what gene therapy could deliver and thus may be lured into medical tourism, both desperate patients and also foolish investors, and all the while charlatans will profit from peddling promise.

I worry that raising hopes too high too quickly will ultimately cause a backlash against more moderate science.

I also worry that even conventional funding bodies will succumb to understandable pressures to fund translational research prematurely and this will actually waste large amounts of valuable public money.

And I worry about a hysterical reaction that could divide society along political lines with people lining up for or against germline gene therapy based on their political positions or personal beliefs rather than a sober examination of the facts, risks and contexts.

Finally, I worry that the focus on human modification will distract us from other issues, such as the use of CRISPR-mediated gene drives that could be used to eradicate rapidly reproducing organisms such as mosquitoes, and could thus be used for both great good or great harm.

But I don't feel the burden of worry too much because I know that, as a scientist, I can and should share the weight of my concerns with society.

Explore further: With stringent oversight, heritable human genome editing could be allowed: report

This article was originally published on The Conversation. Read the original article.

Clinical trials for genome editing of the human germline - adding, removing, or replacing DNA base pairs in gametes or early embryos - could be permitted in the future, but only for serious conditions under stringent oversight, ...

Recent evidence demonstrating the feasibility of using novel CRISPR/Cas9 gene editing technology to make targeted changes in the DNA of human embryos is forcing researchers, clinicians, and ethicists to revisit the highly ...

The National Academy of Sciences and National Academy of Medicine issued a 258-page report Tuesday (Feb. 14) focused on human genome editing. It lays out principles and recommendations for the U.S. government and governments ...

At the conclusion of the recent International Summit on Human Gene Editing in Washington, DC, its organizing committee released a much-anticipated statement recommending how human genetic engineering should be regulated. ...

Don't expect designer babies any time soonbut a major new ethics report leaves open the possibility of one day altering human heredity to fight genetic diseases, with stringent oversight, using new tools that precisely ...

Personalized medicine, which involves tailoring health care to each person's unique genetic makeup, has the potential to transform how we diagnose, prevent and treat disease. After all, no two people are alike. Mapping a ...

Work on gene therapy is showing significant progress for restoring muscle strength and prolonging lives in dogs with a previously incurable, inherited neuromuscular disease. UW Medicine Institute for Stem Cell and Regenerative ...

A genomic study of baldness identified more than 200 genetic regions involved in this common but potentially embarrassing condition. These genetic variants could be used to predict a man's chance of severe hair loss. The ...

Purdue University and Indiana University School of Medicine scientists were able to force an epigenetic reaction that turns on and off a gene known to determine the fate of the neural stem cells, a finding that could lead ...

Just before Rare Disease Day 2017, a study from the Monell Center and collaborating institutions provides new insight into the causes of trimethylaminura (TMAU), a genetically-transmitted metabolic disorder that leads to ...

Monash University and Danish researchers have discovered a gene in worms that could help break the cycle of overeating and under-exercising that can lead to obesity.

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Human genome editing report strikes the right balance between risks and benefits - Medical Xpress

1102 Bates Street, Suite 1670

Houston, Texas 77030

The GMP Facilities at the Center for Cell and Gene Therapy at Baylor College of Medicine are among the largest and most modern in the United States. Manufacturing of therapeutic biologics has existed at Baylor for more than twenty years, and is now centralized in 19,000 square feet on the 16th floor of the Feigin Center at Texas Childrens Hospital.

This facility opened in 2010 and contains 22 ISO 7 clean room suites supported by dedicated space for cell and product cold storage, flow cytometric analysis, quality control testing, data management and storage and quality assurance activities. The staff has extensive experience in GMP manufacturing of a wide variety of products and intermediates for cellular therapies and of viral and non-viral vectors, and master and working cell banks.

Manufacturing and testing supports more than 30 investigator-sponsored INDs conducted at the Texas Medical Center and institutions around the United States. Products have also been prepared under contract for researchers in Europe, Asia and Australia. The range of products that have been manufactured is shown under the Vector and Cell Therapy Facilitypages.

The facility is also accredited by the Foundation for the Accreditation of Cellular Therapy (FACT)and is CLIA registered for high complexity testing.

Center for Cell & Gene Therapy - cGMP Facilities

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Center For Cell & Gene Therapy - Cell Therapy

An orthopedist orders special testing to determine if an elder patient with right hip pain which limits walking and driving might need surgery to improve their quality of life.

Physicians are rigorously trained to make decisions in the best interest of their patients. Even after medical school and residency, doctors must follow the challenges of evidence-based medicine, standard of care, peer review, and muster the time for continuing medical education and certification.

Doctrors are not only held accountable by their peers, but also legally as they could be subject to lawsuits. Additionally, state licensing agencies overseeing medical professionals can discipline them should they not practice medicine up to the standards of quality medical decision-making.

However, what if the teens pediatrician feels hospitalization is acutely needed for mental illness, but it is denied by the insurance company? What if the Workers Comp physician orders an MRI for the powerline workers ailing right arm, but it is denied? Or, if special testing to evaluate grandmas worsening mobility and pain is turned down by the HMO? Who is held accountable?

To justify requests for specific patient care, physicians are forced to have Peer-to-Peer phone discussions with doctors employed by insurance companies, Workers Comp, and HMOs. Frequently, these conversations result in denial of further care without medical justification. A controversial question arises: Are denials by these company doctors considered a medical decision?

They are not. These decisions are considered utilization review. What does this mean? They are making decisions based on controlling costs, which is in the financial interest of the for-profit agencies they serve, but not necessarily in the best interest of the patient. Even though they are licensed doctors practicing medicine, their role in patient care is under the guise of utilization review, and therefore not under the scrutiny of state licensing agencies.

What if these physicians deny care because they are incentivized to enhance personal bonuses? More so, what if some are making decisions outside the realm of their medical expertise (e.g. a urologist on a diabetic)? Who holds these physicians accountable for moral transgressions, or lack of judgement?

In California, we have a Medical Board which oversees licensing for all state physicians. If you report a licensed physician for making substandard medical decisions, an investigation ensues. If though the doctor is employed by an insurance company, Workers Comp, or HMO and makes denial decisions on their behalf, it is considered utilization review and they are not held accountable.

I do not pretend to understand every law and rule governing the Medical Board. But these companies have created legal barriers protecting doctors who might make substandard medical decisions.

Many physicians continue to fight for patient care rights despite frustration and helplessness of ongoing phone calls and paperwork they face. Yet substandard medical care will hamper their efforts as laws are manipulated and oversight is negligible.

Making medical decisions has never been easy. Assuring accountability makes it even harder.

Gene Uzawa Dorio, M.D.

Read more from the original source:
Dr. Gene Dorio: Making Medical Decisions Without Accountability - KHTS Radio

When Nisa Leung was pregnant with her first child in 2012, her doctor in Hong Kong offered her a choice. She could take a prenatal test that would require inserting a needle into her uterus, or pay $130 more for an exam that would draw a little blood from her arm.

Leung opted for the simpler and less risky test, which analyzed bits of the babys DNA that had made its way into her bloodstream. Then, Leung went on to do what she often does when she recognizes a good product: look around for companies to invest in.

The managing partner at Qiming Venture Partners decided to put money into Chinese genetic testing firm Berry Genomics, which eventually entered into a partnership with the Hong Kong-based inventor of the blood test. Over the next few months, Berry is expected to be absorbed into a Chinese developer in a 4.3 billion yuan ($625 million) reverse merger. And Leungs venture capital firm would be the latest to benefit from a boom in so-called precision medicine, an emerging field that includes everything from genetic prenatal tests to customizing treatments for cancer patients.

Source: Qiming Venture Partners

China has made the precision medicine field a focus of its 13th five-year plan, and its companies have been embarking on ambitious efforts to collect a vast trove of genetic and health data, researching how to identify cancer markers in blood, and launching consumer technologies that aim to tap potentially life-saving information. The push offers insight into Chinas growing ambitions in science and biotechnology, areas where it has traditionally lagged developed nations like the U.S.

Investing in precision medicine is definitely the trend, said Leung,whos led investments in more than 60 Chinese health-care companies in the past decade. As China eyes becoming a biotechnology powerhouse globally, this is an area we will venture into for sure and hopefully be at the forefront globally.

New Chinese firms like iCarbonX and WuXi NextCode that offer consumers ways to learn more about their bodies through clues from their genetic make up are gaining popularity. Chinese entrepreneurs and scientists are also aiming to dominate the market for complex new procedures like liquid biopsy tests, which would allow for cancer testing through key indicators in the blood.

iCarbonX founder Wang Jun.

Photographer: Calvin Sit/Bloomberg

Such research efforts are still in early stages worldwide. But doctors see a future beyond basic commercial applications, aiming instead for drugs and treatment plans tailored to a persons unique genetic code and environmental exposure, such as diet and infections.

Isaac Kohane, a bioinformatics professor at Harvard University, says when it comes to precision medicine, the science community has Google maps envy. Just as the search engine has transformed the notion of geography by adding restaurants, weather and other locators,more details on patients can give doctors a better picture on how to treat diseases.

For cancer patients, for example, precision medicine might allow oncologists to spot specific mutations in a tumor. For many people with rare ailments like muscle diseases or those that cause seizures, it allows for earlier diagnosis. Pregnant women, using the kind of tests that Leung used, could also learn more about the potential for a child to inherit a genetic disease.

The global interest in the field comes as the cost of sequencing DNA, or analyzing genetic information, is falling sharply. But a number of hurdles remain. Relying on just genes isnt enough, and there must also be background information on a patients lifestyle and medication history.

Precision medicine applications also require heavy investment to store large amounts of information. A whole genome is over 100 gigabytes, according toan e-mailed response to questions from Edward Farmer, WuXi NextCodes vice-president of communications and new ventures. So you can imagine that analyzing thousands or hundreds of thousands of genomes is a true big data challenge."

WuXi NextCode was formed after Shanghai-based contract research giant WuXi AppTec Inc. acquired genomic analysis firm NextCode Health, a spin-off from Reykjavik, Iceland-based Decode Genetics, which has databases on the islands population. Wuxi NextCode continues to have an office in Iceland, where the population is relatively homogenous and therefore good for gene discovery.

Source: WuXi NextCODE

"Genomics today is like the computer industry in the 70s," said Hannes Smarason, WuXi NextCodes co-founder and chief operating officer. "Weve made great progress but theres still a long way to go.

In China, Wuxi NextCode now offers consumers genetic tests that cost between about 2,500 yuan and 8,000 yuan, providing more details on rare conditions a child might be suffering from or even the risk of passing on an inherited disease.

China is diverse and with 1.4 billion people, the planets most populous nation. WuXi NextCode announced a partnership with Huawei Technologies Co.,Chinas largest telecommunications equipment maker, in May to enable different institutions and researchers to store their data.

The goal is to use that deep pool of information -- which ranges from genome sequences to treatment regimens -- to find more clues on tackling diseases. WuXi says that this will in many instances enable the largest studies ever undertaken in many diseases.

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The global precision medicine market was estimated to be worth $56 billion in revenue at the end of 2016,with China holding about 4 to 8 percent of the global market, according to a December report from Persistence Market Research.

Encouraging interventions for some patients too early, even before they have life-threatening diseases, comes with risks and ethical questions, Laura Nelson Carney,an analyst at Sanford C Bernstein, wrote in a Jan. 6 note. Still, precision medicine research has many benefits, and some in China see the countrys push as a significant opportunity "to scientifically leapfrog the West, she said.

In the U.S., universities, the National Institutes of Health and American drugmakers are part of a broad march into precision medicine.

Amgen Inc. bought Icelandic biotechnology company DeCode Genetics for $415 million in 2012, to acquire its massive database on Icelands population. U.S.-based Genentech Inc. is collaborating with Silicon Valley startup 23andMe to study the genetic underpinnings of Parkinsons disease.

Humans are computable," saidWang Jun, the chief executive officer of ChinasiCarbonX. "So we need a computable model that we can use to intervene and change peoples status, thats the whole point.

Read more from the original source:
China Turns to Precision Medicine in Fight Against Cancer ... - Bloomberg