Archive for the ‘Gene Therapy Research’ Category

Scientists in Australia claim to have discovered what could be a life-long cure for potentially fatal allergies to peanuts, shellfish and other food.

The researchers said they had been able to turn off the allergic response in tests on mice using gene therapy to desensitise the bodys immune system, and suggested this could also be used to treat asthma.

They predicted human trials could begin in just five or six years.

Commenting on the study, a leading British expert said scientists had managed to cure allergies in mice before without this leading to an effective human treatment, but added that the new research could lead to the "Holy Grail" of allergy treatment.

He was sceptical about the researchers' claims their technique might be effective against asthma, but Asthma UK said it was "a very exciting step forward".

Allergies occur when the immune system over-reacts to something that is usually harmless. In the journal JCI Insight, the Australian researchers reported they had used genetic techniques to prevent this from happening in mice who were allergic to the protein in egg whites.

In a video about the new research, Professor Ray Steptoe, of Queensland University, said: We can actually turn off the response. What that means is the disease is stopped in its tracks.

What we do is we stop the underlying disease that causes these symptoms. That could revolutionise treatment for severe allergies. It would prevent, we think, some of the life-threatening allergic episodes that occur for people who are allergic to foods for instance.

That would make a huge difference for people with severe allergies what that would mean is they would no longer be in fear of life-threatening incidents if they were to go to a restaurant and be exposed to shellfish and they werent aware that was in the food.

Kids with peanut allergies could go to school without any fear of being contaminated from other kids food.

We envisage in the future, with this approach, that they could go to the doctors rooms, get a single treatment and that would give them permanent protection from future allergic attacks or asthma attacks.

He added that the researchers hoped human trials could begin in five to six years, estimated it would take a similar period after that for the treatment to be available to patients.

Professor Adnan Custovic, an allergy expert at Imperial College London, expressed particular caution about the claim the treatment would be effective against asthma as the condition is caused by a completely different mechanism to the one behind food allergies.

But he added: This is one of the potentially exciting approaches to treating allergies.

Its sort of approach, where you try to switch off the allergic response, is kind of the Holy Grail, but a mouse model is not the same as a human model.

We can cure allergies in mice but we cannot do it in humans the mechanisms are not identical. Only time will tell whether this approach will be a viable one.

And he criticised the degree of optimism about the technique expressed by the Australian team.

My real problem with this sort of bombastic statements like this is people with asthma it gives them hope which very often is not realistic, Professor Custovic said.

However Dr Erika Kennington, head of research at Asthma UK, was more optimistic.

This is potentially a very exciting step forward in asthma research," she said.

"Allergen immunotherapy exposing people to small amounts of an allergen in order to build up tolerance is currently the only disease-altering treatment available for asthma but it can have significant side effects in some people, and every other existing asthma treatment and medication works by reducing or relieving the symptoms.

"These findings suggesting a novel approach to reversing allergic disease are therefore very welcome.

We also know that there are certain allergy triggers that cause asthma flare ups, which makes this research important in possibly reducing the risk of life-threatening asthma attacks."

But Dr Kennington also pointed to the difference between animal and human trials.

A lot more research is needed to see if the same results can be achieved in people before we can say that a cure for asthma is around the corner," she said.

In the study of the allergic mice, the researchers inserted a gene into blood stem cells that controls the immune response to the egg white.

The genetically modified cells were then injected into the mices bone marrow, where they produced new blood cells that were able to turn off the allergic response.

The researchers hope to create a similar form of gene therapy that works on humans after a single injection.

We havent quite got it to the point where its as simple as getting a flu jab, so we are working on making it simpler and safer so it could be used across a wide cross-section of affected individuals, Dr Steptoe said.

Dr Louisa James, British Society for Immunology spokesperson and an immunologistat Queen Mary University of London, said allergies were "far more complex than can be replicated in an animal model".

"Patients with severe allergies often react to several different types of allergen and symptoms can develop over several years," she said.

"Although the results are encouraging and heading in the right direction, it is too early to predict whether this form of therapy could ever be used to treat allergies in humans.

"As the authors state in their paper 'gene-therapy is not yet suitable for clinical application to mild disease in young individuals'.

"There are simply too many open questions around the translation of these findings from animal models into humans.Would the cells engineered to produce allergens produce the same response in humans? How would other immune cells that play a critical role in human allergy be affected? What are the mechanisms that switch off the immune response and are they comparable in humans?

This approach holds promise, and further research is certainly warranted, but claims that a single injection could switch off allergies are over-optimistic at this time.

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Allergy treatment: Scientists claim breakthrough that could lead to ... - The Independent

A consultant studies a mammogram. The drug olaparib could slow cancer growth by three months, researchers have found. Photograph: Rui Vieira/PA

A type of inherited and incurable breast cancer that tends to affect younger women could be targeted by a new gene therapy, researchers have found.

A small study presented at the worlds largest cancer conference found treating patients with the drug olaparib could slow cancer growth by three months and be less toxic for patients with inherited BRCA-related breast cancer.

Researchers said there was not enough data to say whether patients survived longer as a result of the treatment.

We are in our infancy, said Dr Daniel Hayes, president of the American Society of Clinical Oncology and professor of breast cancer research at the University of Michigan. This is clearly an advance; this is clearly proof of concept these can work with breast cancer.

Does it look like its going to extend life? We dont know yet, he said.

The drug is part of the developing field of precision medicine, which targets patients genes to tailor treatment.

It is a perfect example of how understanding a patients genetics and the biology of their tumor can be used to target its weaknesses and personalize treatment, said Andrew Tutt, director of the Breast Cancer Now Research Centre at The Institute of Cancer Research.

Olaparib is already available for women with BRCA-mutant advanced ovarian cancer, and is the first drug to be approved that is directed against an inherited genetic mutation. The study was the first to show olaparib can slow growth of inherited BRCA-related breast cancer. The drug is not yet approved for that use.

People with inherited mutations in the BRCA gene make up about 3% of all breast cancer patients, and tend to be younger. The median age of women in the olaparib trial was 44 years old.

BRCA genes are part of a pathway to keep cells reproducing normally. An inherited defect can fail to stop abnormal growth, thus increasing the risk of cancer. The study examined the effectiveness of olaparib against a class of BRCA-related cancers called triple negative. Olaparib is part of a class of four drugs called PARP-inhibitors that work by shutting down a pathway cancer cells use to reproduce.

The study from Memorial Sloan Kettering Cancer Center in New York randomly treated 300 women with advanced, BRCA-mutated cancer with olaparib or chemotherapy. All the participants had already received two rounds of chemotherapy.

About 60% of patients who received olaparib saw tumors shrink, compared with 29% of patients who received chemotherapy. That meant patients who received olaparib saw cancer advance in seven months, versus four months for only chemotherapy.

Researchers cautioned it is unclear whether olaparib extended life for these patients, and that more research was needed to find out which subset of patients benefit most from olaparib.

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New gene therapy offers hope against incurable form of breast cancer - The Guardian

Posted June 03, 2017 08:59:49

Sufferers of severe allergies like asthma and lethal food allergies could gain lifetime relief in a single treatment, according to scientists at the University of Queensland.

The researchers believe a simple injection that will "turn off" the immune response that causes allergic reactions in affected people could be available within 10 years.

Immunologist Associate Professor Ray Steptoe said it was a "major breakthrough".

"Learning how to turn off this immune response has been challenge for immunotherapy for a long time," Dr Steptoe said.

"We haven't quite got it to the point where it's as simple as getting a flu jab, so we are working on making it simpler and safer so it could be used across a wide cross-section of affected individuals."

Dr Steptoe said allergic symptoms, such as breathing difficulties, occur when immune cells known as T-cells react to the protein in allergens.

"The challenge is that these T-cells develop a form of immune 'memory' and become very resistant to treatments," he said.

The researchers worked with an asthma allergen and used gene therapy to break the damaging cycle.

"We have now been able 'wipe' the memory of these T-cells in animals with gene therapy, de-sensitising the immune system so that it tolerates the protein," Dr Steptoe said.

"The research could be applied treat those who have severe allergies to peanuts, bee venom, shell fish and the like."

Importantly, the therapy specifically targets the memory for the allergen protein, while leaving other immune responses unaffected.

"It does it in a highly targeted way, without turning off the memory that is protective such as the important immune response you get from vaccinations, the ones that protect you from the flu and other infectious diseases we're vaccinated against," he said.

The research findings have been welcomed by the Asthma Foundation of Queensland and New South Wales, which said it looked forward to the development of future treatments.

CEO Dr Peter Anderson said more than one million Australians suffered with asthma symptoms.

"The Foundation welcomes the findings of this research and looks forward to a day in the future when a safe one-off treatment may be available that has the potential to eliminate any experience of asthma in vulnerable patients," he said.

Topics: health, diseases-and-disorders, allergies, medical-research, qld, brisbane-4000

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Allergy research 'breakthrough' could lead to treatment in a decade ... - ABC Online

Scientists have used gene therapy to 'switch off' the immune response that causes asthma, and are hopeful that the same technique could be used to target other severe allergies to peanuts, bee venom, and shellfish,keeping them at bay for life.

The research, which has so far seen success in animal trials, works byerasing the memory of the cells responsible for causing an allergic reaction, and if replicated in humans, could offer a one-off treatment for allergy patients.

"The challenge in asthma and allergies is that these immune cells, known as T-cells, develop a form of immune 'memory', and become very resistant to treatments,"says lead researcherRay Steptoefrom the University of Queensland (UQ) in Australia.

"We have now been able 'wipe' the memory of these T-cells in animals with gene therapy, de-sensitising the immune system so that it tolerates the [allergen] protein."

An allergic response is a hypersensitive immune reaction to a substance that is normally harmless. When people are exposed to their allergic trigger, it can cause anything from itchy eyes and a runny nose to - in the most extreme cases -death.

Asthma is a common allergic response of the airways affecting 2.5 million Australiansand hundreds of millions around the world. About 80 percent of people who experience asthma in Australia are susceptible to hay fever - an allergic response to rye grass pollen.

"When someone has an allergy or asthma flare-up, the symptoms they experience results from immune cells reacting to protein in the allergen," says lead researcherRay Steptoefrom the University of Queensland (UQ) in Australia.

While previous research has looked into using nanoparticle 'trojan horses' to smuggle the allergen past the immune system, and at new immunotherapy approaches, right now, the most effective treatment for people suffering from allergies is to simply avoid all known triggers.

To figure out a better way, Steptoe and his teamtook bone marrow from mice that had been genetically modified to have a resistance against asthma caused by rye grass pollen, and transplanted the bone marrow into unmodified mice.

"We take blood stem cells, insert a gene which regulates the allergen protein, and we put that into the recipient," says Steptoe.

"Those engineered cells produce new blood cells programmed to express the protein and target specific immune cells, which 'turn off' the allergic response."

Even though this study only looked at asthma, the researchers hope that the same approach could be used to provide protection against other common allergies - food and otherwise.

"Our work used an experimental asthma allergen, but this research could be applied to treat those who have severe allergies to peanuts, bee venom, shellfish and the like," Steptoe said.

But before we start throwing our puffers in the bin, the studies still have to be replicated in human trials, and that's where things get much more complicated.

"In the real world, unfortunately, it's not just usually a single allergen protein [that causes an immune response]. There might be several proteins that you might be allergic to and you'd have to target each of those proteins," Steptoe told ScienceAlert.

"We're currently doing experiments to see if we can turn off multiple response at the same time."

The research is published in JCI Insight.

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Gene therapy has been used to 'switch off' asthma symptoms - ScienceAlert

(THE CONVERSATION) A new research paper is stirring up controversy among scientists interested in using DNA editing to treat disease.

In a two-page article published in the journal Nature Methods on May 30, a group of six scientists report an alarming number of so-called off-target mutations in mice that underwent an experimental gene repair therapy.

CRISPR, the hot new gene-editing technique thats taken biology by storm, is no stranger to headlines. What is unusual, however, is a scientific article so clearly describing a potentially fatal shortcoming of this promising technology.

The research community is digesting this news with many experts suggesting flaws with the experiment, not the revolutionary technique.

The research team sought to repair a genetic mutation known to cause a form of blindness in mice. This could be accomplished, they showed, by changing just one DNA letter in the mouse genome.

They were able to successfully correct the targeted mutation in each of the two mice they treated. But they also observed an alarming number of additional DNA changes more than 1,600 per mouse in areas of the genome they did not intend to modify.

The authors attribute these unintended mutations to the experimental CRISPR-based gene editing therapy they used.

A central promise of CRISPR-based gene editing is its ability to pinpoint particular genes. But if this technology produces dangerous side effects by creating unexpected and unwanted mutations across the genome, that could hamper or even derail many of its applications.

Several previous research articles have reported off-target effects of CRISPR, but far fewer than this group found.

The publicly traded biotech companies seeking to commercialize CRISPR-based gene therapies Editas Medicine, Intellia Therapeutics and Crispr Therapeutics all took immediate stock market hits based on the news.

Experts in the field quickly responded.

Either the enzyme is acting at near optimal efficiency or something fishy is going on here, tweeted Matthew Taliaferro, a postdoctoral fellow at MIT who studies gene expression and genetic disease.

The Cas9 enzyme in the CRISPR system is what actually cuts DNA, leading to genetic changes. Unusually high levels of enzyme activity could account for the observed off-target mutations more cutting equals more chances for the cell to mutate its DNA. Different labs use slightly different methods to try to ensure the right amount of cuts happen only where intended.

Unusual methods were used, tweeted Lluis Montoliu, who runs a lab at the Spanish National Centre for Biotechnology that specializes in editing mice genes using CRISPR. He believes the authors used suboptimal molecular components in their injected CRISPR therapies specifically a plasmid that causes cells to produce too much Cas9 enzyme likely leading to the off-target effects they observed.

Gatan Burgio, whose laboratory at the Australian National University is working to understand the role that cellular context plays on CRISPR efficiency, believes the papers central claim that CRISPR caused such an alarming number of off-target mutations is not substantiated.

Burgio says there could be a range of reasons for seeing so many unexpected changes in the mice, including problems with accurately detecting DNA variation, the extremely small number of mice used, random events happening after Cas9 acted or, he concedes, problems with CRISPR itself.

Burgio has been editing the DNA of mice using CRISPR since 2014 and has never seen a comparable level of off-target mutation. He says hes confident that additional research will refute these recent findings.

Although the news of this two-mouse experiment fired up the science-focused parts of the Twittersphere, the issue it raises is not new to the field.

Researchers have known for a few years now that off-target mutations are likely given certain CRISPR protocols. More precise variants of the Cas9 enzyme have been shown to improve targeting in human tissue the lab.

Researchers have also focused on developing methods to more efficiently locate off-target mutations in the animals they study.

As scientists continue to hone the gene-editing technique, we recognize theres still a way to go before CRISPR will be ready for safe and effective gene therapy in humans.

This article was originally published on The Conversation. Read the original article here: http://theconversation.com/crispr-controversy-raises-questions-about-gene-editing-technique-78638.

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CRISPR controversy raises questions about gene-editing technique - San Francisco Chronicle

Xconomy Texas

San Antonio Most diabetes treatments work by giving the body the insulin it needs to break down sugar. But that approach deals with the symptoms of diabetes. In recent years, scientists and companies have taken aim at the root cause of the condition by attempting to stimulate or replace the cells in the pancreas responsible for producing insulin in the first place. One of them is a San Antonio researcher hoping to use gene therapya potentially one-time, long lasting treatmentto do the trick.

When cells in the pancreas, known as beta cells, either get destroyed by the immune system or stop producing enough insulin, the result is type 1 or type 2 diabetes. Companies large and small-from European diabetes drug giant Novo Nordisk to privately held startups ViaCyte, of San Diego, and Semma Therapeutics, of Cambridge, MAwant to engineer stem cells that develop into pancreatic beta cells to help a patient produce insulin.

Other researchers, such as Bruno Doiron, a scientist and assistant professor at the University of Texas Health Science Center at San Antonio, have different ideas. Doiron has developed an injectible treatment consisting of three molecules glucokinase, a second that targets a protein known as PTP1B, and a third that targets a protein called Pdx-1, a so-called transcription factor that regulates genesthat, when infused into the body, are meant to help stimulate the formation of new beta cells. Doiron has tried the method on mice, and based on some encouraging early results, intends to move the work forward through a startup company.

You have to prove you can translate that to a large animal model, he says.

The San Antonio company, Syner-III, got its name because of the synergistic use of three molecules to generate the beta cells, he says. Those molecules are administered via a gene therapy procedure: theyre stuffed into a modified virus and injected directly into the pancreas in a one-time treatment, where they are meant to stimulate beta cell production. The work was published in the peer-reviewed journal Current Pharmaceutical Biotechnology in 2016.

Doiron hopes to raise as much as $10 million to complete preclinical testing.

Others, including Novartis, are considering different ways of boosting beta cell production. Researchers from the Swiss company published findings in Nature Communications that showed a group of compounds called aminopyrazines could be packed into a pill and similarly lead to more beta cells, and more insulin, in mice. Such attempts are fraught with failure, however. In an article on its own website, Novartis notes that researchers have succeeded in producing beta cells in mice many times, but havent been able to reproduce those results in humans.

The potential payoff, however, is huge. Some 29.1 million Americans have diabetes, and 1.25 million of them have type 1 diabetes, according to the American Diabetes Association. Doiron believes the therapy may be able to help both types. While stem cell research has had its share of failures and competition continues to increase in insulin therapysuch as pumps that automatically deliver the treatmentDoiron says a gene therapy, if successful, could result in a longer-lasting, more effective treatment.

When I use your own body to produce medicine, that drastically changes the field, he says.

David Holley is Xconomy's national correspondent based in Austin, TX. You can reach him at dholley@xconomy.com

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With Gene Therapy for Diabetes, San Antonio Researcher Eyes Funding - Xconomy

The oncologist was blunt: Stefanie Johos colon cancer was raging out of control and there was nothing more she could do. Flanked by her parents and sister, the 23-year-old felt something wet on her shoulder. She looked up to see her father weeping.

I felt dead inside, utterly demoralized, ready to be done, Joho remembers.

But her younger sister couldnt accept that. When the family got back to Johos apartment in New Yorks Flatiron district, Jess opened her laptop and began searching frantically for clinical trials, using medical words shed heard but not fully understood. An hour later, she came into her sisters room and showed her what shed found. Im not letting you give up, she told Stefanie. This is not the end.

That search led to a contact at Johns Hopkins University, and a few days later, Joho got a call from a cancer geneticist co-leading a study there. Get down here as fast as you can! Luis Diaz said. We are having tremendous success with patients like you.

What followed is an illuminating tale of how one womans intersection with experimental research helped open a new frontier in cancer treatment with approval of a drug that, for the first time, capitalizes on a genetic feature in a tumor rather than on the diseases location in the body.

The breakthrough, made official last week by the Food and Drug Administration, immediately could benefit some patients with certain kinds of advanced cancer that arent responding to chemotherapy. Each should be tested for that genetic signature, scientists stress.

These are people facing death sentences, said Hopkins geneticist Bert Vogelstein. This treatment might keep some of them in remission for a long time.

In August 2014, Joho stumbled into Hopkins for her first infusion of the immunotherapy drug Keytruda. She was in agony from a malignant mass in her midsection, and even with the copious amounts of oxycodone she was swallowing, she needed a new fentanyl patch on her arm every 48 hours. Yet within just days, the excruciating back pain had eased. Then an unfamiliar sensation hunger returned. She burst into tears when she realized what it was.

As months went by, her tumor shrank and ultimately disappeared. She stopped treatment this past August, free from all signs of disease.

[Negotiating cancer: Tips from one whos done it ]

The small trial in Baltimore was pivotal, and not only for the young marketing professional. It showed that immunotherapy could attack colon and other cancers thought to be unstoppable. The key was their tumors genetic defect, known as mismatch repair (MMR) deficiency akin to a missing spell-check on their DNA. As the DNA copies itself, the abnormality prevents any errors from being fixed. In the cancer cells, that means huge numbers of mutations that are good targets for immunotherapy.

The treatment approach isnt a panacea, however. The glitch under scrutiny which can arise spontaneously or be inherited is found in just 4percent of cancers overall. But bore in on a few specific types, and the scenario changes dramatically. The problem occurs in up to 20percent of colon cancers and about 40percent of endometrial malignancies cancer in the lining of the uterus.

In the United States, researchers estimate that initially about 15,000 people with the defect may be helped by this immunotherapy. That number is likely to rise sharply as doctors begin using it earlier on eligible patients.

Joho was among the first.

***

Even before Joho got sick, cancer had cast a long shadow on her family. Her mother has Lynch syndrome, a hereditary disorder that sharply raises the risk of certain cancers, and since 2003, Priscilla Joho has suffered colon cancer, uterine cancer and squamous cell carcinoma of the skin.

Stefanies older sister, Vanessa, had already tested positive for Lynch syndrome, and Stefanie planned to get tested when she turned 25. But at 22, several months after she graduated from New York University, she began feeling unusually tired. She blamed the fatigue on her demanding job. Her primary-care physician, aware of her mothers medical history, ordered a colonoscopy.

When Joho woke up from the procedure, the gastroenterologist looked like a ghost, she said. A subsequent CT scan revealed a very large tumor in her colon. Shed definitely inherited Lynch syndrome.

She underwent surgery in January 2013 at Philadelphias Fox Chase Cancer Center, where her mother had been treated. The news was good: The cancer didnt appear to have spread, so she could skip chemotherapy and follow up with scans every three months.

[More than two-thirds of cancer mutations are due to random DNA copying errors, study says]

By August of that year, though, Joho started having relentless back pain. Tests detected the invasive tumor in her abdomen. Another operation, and now she started chemo. Once again, in spring 2014, the cancer roared back. Her doctors in New York, where she now was living, switched to a more aggressive chemo regimen.

This thing is going to kill me, Joho remembered thinking. It was eating me alive.

She made it to Jesss college graduation in Vermont that May. Midsummer, her oncologist confessed he was out of options. As he left the examining room, he mentioned offhandedly that some interesting work was going on in immunotherapy. But when Joho met with a hospital immunologist, that doctor told her no suitable trials were available.

Joho began planning to move to her parents home in suburban Philadelphia: I thought, Im dying, and Id like to breathe fresh air and be around the green and the trees.

Her younger sister wasnt ready for her to give up. Jess searched for clinical trials, typing in immunotherapy and other terms shed heard the doctors use. Up popped a trial at Hopkins, where doctors were testing a drug called pembrolizumab.

***

Pembro is part of a class of new medications called checkpoint inhibitors that disable the brakes that keep the immune system from attacking tumors. In September 2014, the treatment was approved by the FDA for advanced melanoma and marketed as Keytruda. The medication made headlines in 2015 when it helped treat former president Jimmy Carter for melanoma that had spread to his brain and liver. It later was cleared for several other malignancies.

Yet researchers still dont know why immunotherapy, once hailed as a game changer, works in only a minority of patients. Figuring that out is important for clinical as well as financial reasons. Keytruda, for example, costs about $150,000 a year.

By the time Joho arrived at Hopkins, the trial had been underway for a year. While an earlier study had shown a similar immunotherapy drug to be effective for a significant proportion of patients with advanced melanoma or lung or kidney cancer, checkpoint inhibitors werent making headway with colon cancer. A single patient out of 20 had responded in a couple of trials.

Why did some tumors shrink and others didnt? What was different about the single colon cancer patient who benefited?

Drew Pardoll, director of the Bloomberg-Kimmel Institute for Cancer Immunotherapy at Hopkins, and top researcher Suzanne L. Topalian took the unusual step of consulting with the cancer geneticists who worked one floor up.

This was the first date in what became the marriage of cancer genetics and cancer immunology, Pardoll said.

[A consumers guide to the hottest field in cancer treatments immunotherapy]

In a brainstorming session, the geneticists were quick to offer their theories. They suggested that the melanoma and lung cancer patients had done best because those cancers have lots of mutations, a consequence of exposure to sunlight and cigarette smoke. The mutations produce proteins recognized by the immune system as foreign and ripe for attack, and the drug boosts the systems response.

And that one colon-cancer patient? As Vogelstein recalls, We all said in unison, He must have MMR deficiency! because such a genetic glitch would spawn even more mutations. The abnormality was a familiar subject to Vogelstein, who in the 1990s had co-discovered its role in the development of colon cancer. But the immunologists hadnt thought of it.

When the patients tumor tissue was tested, it was indeed positive for the defect.

The researchers decided to run a small trial, led by Hopkins immunologist Dung Le and geneticist Diaz, to determine whether the defect could predict a patients response to immunotherapy. The pharmaceutical company Merck provided its still-experimental drug pembrolizumab. Three groups of volunteers were recruited: 10 colon cancer patients whose tumors had the genetic problem; 18 colon cancer patients without it; and 7 patients with other malignancies with the defect.

The first results, published in 2015 in the New England Journal of Medicine, were striking. Four out of the 10 colon cancer patients with the defect and 5 out of the other 7cancer patients with the abnormality responded to the drug. In the remaining group, nothing. Since then, updated numbers have reinforced that a high proportion of patients with the genetic feature benefit from the drug, often for a lengthy period. Other trials by pharmaceutical companies have shown similar results.

The Hopkins investigators found that tumors with the defect had, on average, 1,700 mutations, compared with only 70 for tumors without the problem. That confirmed the theory that high numbers of mutations make it more likely the immune system will recognize and attack cancer if it gets assistance from immunotherapy.

The studies were the foundation of the FDAs decision on Tuesday to green-light Keytruda to treat cancers such as Johos, meaning malignancies with certain molecular characteristics. This first-ever site-agnostic approval by the agency signals an emerging field of precision immunotherapy, Pardoll said, one in which genetic details are used to anticipate who will respond to treatments.

***

For Joho, now 27 and living in suburban Philadelphia, the hard lesson from the past few years is clear: The cancer field is changing so rapidly that patients cant rely on their doctors to find them the best treatments. Oncologists can barely keep up, she said. My sister found a trial I was a perfect candidate for, and my doctors didnt even know it existed.

Her first several weeks on the trial were rough, with an early hospitalization after she cut back too quickly on her fentanyl and went into withdrawal. She still has some lasting side effects today joint pain in her knees, minor nausea and fatigue but they are manageable.

I have had to adapt to some new limits, she acknowledged. But I still feel better than I have in five years.

The FDAs decision last week was an emotional moment. Diaz, now at Memorial Sloan Kettering Cancer Center in New York, immediately texted her. We did it! he exulted.

I got chills all over my body, Joho said. To think that I was at the end of the road, with no options, and then to be part of such a change.

Her experience has prompted her to drop plans to go back into marketing. Now she wants to help patients navigate the new cancer landscape. Become an expert on your cancer is her message. Dont be passive. She encourages patients to try clinical trials.

As a cancer survivor with Lynch syndrome, Joho will be closely watched; if she relapses, she is likely to be treated again with immunotherapy. And if her mother relapses, Keytruda might now be her best chance.

Coming out the other side, I feel really lucky, Joho said. Shes also grateful for something else: A few years ago, her sister Jess was tested for the disorder that has so affected their family. She was negative.

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'This is not the end': Using immunotherapy and a genetic glitch to give cancer patients hope - Washington Post

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While there is considerable optimism among researchers, they have not been able to completely comprehend the pathology of neurodegenerative disorders or distinguish between healthy aging and neurodegeneration at the initial stages of the diseases. The risk of therapeutic failures is also high during early clinical studies of neurological disorders.

Strong therapeutic platforms for AD and PD will eventually allow drug developers to create therapies for all neurodegenerative diseases. The leading regions for AD and PD research are:

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Research on gene therapy for fighting blindness and providing nutritional and social support to elderly both nominated for US-Ireland research innovation award

By Sarah Meehan on Tuesday, May 30, 2017 Leave a Comment

Professor Jane Farrar from Trinitys School of Genetics and Microbiology and Professor Sabina Brennan a research assistant from Trinitys Institute of Neuroscience, have been nominated for a US-Ireland research innovation award. Both professors are shortlisted in the Higher Education Institution category.

The awards are a joint initiative between the Royal Irish Academy and the American Chamber of Commerce Ireland, and award ideas originating in Irish organisations that make a social and economic impact through research innovation in meeting market needs. The awards are presented in three categories: an Irish higher education institution or research institution with links to the US corporate sector in Ireland, an Irish start up with USlinks and the corporate award for innovation in the Irish operations of a US company.

Professor Farrar, from Trinitys School of Genetics and Microbiology, was nominated for developing gene therapies for treating inherited blindness. Professor Farrars development of GenableTechnologies, was acquired by Spark Therapeutics Inc, a global leader in gene therapy, based in the United States.

Speaking to Trinity News, Professor Farrar said: We were really delighted to be shortlisted in the HEI category for an Innovation Award this year. We are delighted that Spark Therapeutics, the global leader in gene therapy, in 2016 acquired Genable Technologies, a Trinity company established to expedite development of gene therapies for dominantly inherited ocular disorders.

Professor Brennans research focuses on understanding dementia risk and protective factors to establish how a decline in cognitive function might be prevented or delayed. ReLAte is a mealtime service that tries to tackle social isolation and malnutrition in older adults. The research was funded by Home Instead Senior Care Inc., a US care provider, to investigate the relationship- based nutritional intervention in older adults living at alone.

Professor Brennan told Trinity News that: It is a great honour to be shortlisted for the US-Ireland Research Innovation awards. The fact that two of projects from Trinity College Dublin have been nominated is a testament to Trinitys commitment to innovation and research for impact. The RelAte project was delivered by an amazing team of researchers at Trinity College and the research could not have happened without funding from US company Home Instead Senior Care Inc. or without the wonderful participants who gave so freelyof their time.

Dr Goron Elliott of Trinity Research and Innovation said: Trinity is focused on developing industry partnerships with our talented researchers and excellent infrastructure to create economic and societal benefits we are delighted to have two US-Ireland collaborations represented.

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Two Trinity professors nominated for a US-Ireland research innovation award - Trinity News

[When]the House of Representatives passed the American Health Care Act of 2017,DNA Science addressed the possibility of the AHCA forcing pregnant womento carry doomed fetuses to term, the discussion now in the hands of 13 senators[Now]I fear for the treatments for single-gene conditions,both the short-term and available protein-based ones as well as the not-yet-approved gene therapies.

CNN.comonce told the remarkable story of recent college grad Ryan Dant., [who was diagnosed with] a form of mucopolysaccharidosis (MPS) [at age 3]Ryan wasnt expected to survive beyond age 10, but entered a clinical trial for an enzyme replacement therapy (ERT).It won FDA approval in 2003, [but it is very expensive].

The high cost of lifelong frequent infusions or injections of ERT is why the forever fix of a gene therapy is an attractive alternative, even if a booster or two becomes necessary. Gene therapy delivers the DNA instructions for making the missing enzyme. Another reason to seek gene therapy (or editing) is that enzyme infusions dont reach the brain.

Theoretically, gene therapy should be more economical than ERT, once research costs have been recoupedYet the first FDA approval for a gene therapy has yet to happen.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Will Short Term and Long Term Treatments for Single-Gene Diseases Survive?

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Is gene therapy research for single-gene diseases at risk under Trumpcare? - Genetic Literacy Project

XIANGTAN, Hunan, May 25, 2017 /PRNewswire/ -- Incidence of prostate cancer in China is on the rise, cells in the male prostate are overly replicating, creating cancerous growths that leave the patient in severe pain, constant feelings of urination, and a decrease in life expectancy if not treated. Current treatments for prostate cancer include invasive surgeries, radiation, and oral drugs, however, these western remedies often leave patients with adverse side effects (such as hair loss, nausea, weakness, and emotional damage). Medical researchers are now looking for new cancer treatment to treat prostate and other forms of cancer. The 3D prostate treatment based out of the 3D Urology and Prostate Clinic in China is taking this challenge with their research on gene therapy and how genes created can be transferred to the target region. Gene therapy allows for certain genes to be turned on/off creating an enzymatic cascade that can lead to different outcomes, in cancer researchers hope that gene therapy can stop cell growth and induce apoptosis of tumor cells.

Scientists have created novel "suicide genes" that induce apoptosis in vivo. The human body is more complex than test organisms so getting genes to the right location requires a lot of work, simply swallowing a pill will have the suicides genes be destroyed by hydrochloric acid and digestive enzymes in the stomach. To mediate this issue medical researchers are now using injections of suicide genes coupled to vectors. Current day vectors for gene therapy are done through viruses that are tacked with the lab created genes, however, this process leaves patients susceptible to attacks by the virus. Dr. Xinping Song's 3D prostate clinic has been exploring alternative and more natural methods for gene therapy transfer. Vectors investigated by the Dr. Xinping Song include nanoparticles and cationic liposomes, chitosan, and alkaline polysaccharides, their findings showed that these vectors were all highly specific to cancerous tumors and help get the genes to undergo apoptosis into the tumor cells.

Medical research must continue to experiment with different genes and vectors to push to cure prostate and other forms of cancer. Gene therapy can treat certain forms of cancer naturally with no side effects.

Contact:

Alisa Wang

86-186-7321-6429

prostatecure3d(at)gmail(dot)com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/dr-xinping-songs-3d-prostate-treatment-investigates-vectors-for-gene-therapy-in-prostate-cancer-300463869.html

SOURCE Dr. Song's 3D Prostate and 3D Prostatitis Clinic

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Dr. Xinping Song's 3D Prostate Treatment Investigates Vectors for ... - PR Newswire (press release)

Newswise LOS ANGELES Jay R. Lieberman, MD, chair and professor of orthopedic surgery at the Keck School of Medicine of the University of Southern California has received a five-year, $2.2 million grant from the National Institutes of Healths National Institute of Arthritis and Musculoskeletal and Skin Diseases to research gene therapy to enhance repair of extensive bone injuries. Examples of these types of injuries include fractures with extensive bone loss, non-healing fractures, failed spinal fusion and revision of total joint replacement.

Lieberman will genetically manipulate human bone marrow cells to overproduce bone morphogenetic protein (BMP), which is a protein that spurs progenitor cells to produce bone.

There are a number of bone injuries that are very difficult to repair and lack satisfactory solutions, Lieberman says. My goal with this grant is to determine whether genetically modifying human bone marrow cells to overproduce BMP will help heal large bone defects in an animal model and, ultimately, provide a better alternative for repairs in humans.

Liebermans study will determine the efficacy and safety of the gene therapy as well as establish a cellular dose of the genetically manipulated cells that can be scaled up for potential use in humans.

An abstract of the grant, 2R01AR057076-06A1, is available on the NIH RePORTER website.

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ABOUT THE KECK SCHOOL OF MEDICINE OF USC

Founded in 1885, the Keck School of Medicine of USC is among the nations leaders in innovative patient care, scientific discovery, education, and community service. It is part of Keck Medicine of USC, the University of Southern California's medical enterprise, one of only two university-owned academic medical centers in the Los Angeles area. This includes the Keck Medical Center of USC, composed of the Keck Hospital of USC and the USC Norris Cancer Hospital. The two world-class, USC-owned hospitals are staffed by more than 500 physicians who are faculty at the Keck School. The school today has approximately 1,650 full-time faculty members and voluntary faculty of more than 2,400 physicians. These faculty direct the education of approximately 700 medical students and 1,000 students pursuing graduate and post-graduate degrees. The school trains more than 900 resident physicians in more than 50 specialty or subspecialty programs and is the largest educator of physicians practicing in Southern California. Together, the school's faculty and residents serve more than 1.5 million patients each year at Keck Hospital of USC and USC Norris Cancer Hospital, as well as USC-affiliated hospitals Childrens Hospital Los Angeles and Los Angeles County + USC Medical Center. Keck School faculty also conduct research and teach at several research centers and institutes, including the USC Norris Comprehensive Cancer Center, the Zilkha Neurogenetic Institute, the Eli and Edythe Broad Center for Stem Cell Research and Regenerative Medicine at USC, the USC Cardiovascular Thoracic Institute, the USC Roski Eye Institute and the USC Institute of Urology.

In 2017, U.S. News & World Report ranked Keck School of Medicine among the Top 40 medical schools in the country. For more information, go to keck.usc.edu.

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This press release references support by the National Institutes of Health under award number 2R01AR057076-06A1 ($2,284,028 over five years). One hundred percent of the projects funding will be federally funded.

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Keck School of Medicine of USC Receives $2.2 Million NIH Grant to Fund Research on Healing Difficult Bone Injuries - Newswise (press release)

About 1 out of 5 inherited cases of ALS occur due to mutations in SOD1. How these genetic changes contribute to ALS remains unclear. But according to a growing number of studies, these mutations result in the misfolding and aggregation of this metabolic enzyme, contributing to motor neuron toxicity by multiple mechanisms (see Taylor et al., 2016).

Silence, please. Scientists are developing gene therapy strategies for SOD1 ALS that aim to reduce levels of mutant SOD1 in key tissues affected by the disease including the brain and spinal cord. [Image: Rice University under a CC BY 4.0 license.]

A growing number of researchers are developing potential treatment strategies that aim to reduce levels of mutant SOD1 in the CNS in hopes to slow or stop the progression of the disease. One approach, which involves gene therapy, suppresses expression of the SOD1 gene through RNA silencing-based mechanisms. This strategy, according to a 2016 study led by University of Massachusetts Medical Schools Christian Mueller, can extend the lifespan of pre-onset adult SOD1 mice by up to 20% upon intrathecal injection (Borel et al., 2016).

Now, Martine Barkats and Maria Grazia Biferi of the Institute of Myology in Paris, France introduce a new gene therapy approach that increases survival of pre-symptomatic adult SOD1 G93A mice by more than 50%. An independent analysis, led by ALS Therapy Development Institutes Fernando Vieira and commissioned by Prize4Life, confirmed the teams results.

The Institute of Myology team received the Prize4Life $1.0 million USD Avi Kremer ALS Treatment Prize in recognition of reaching this key milestone. This is the best efficacy [observed] in a SOD1 mouse, said Nicole Szlezk, Chairman of Prize4Lifes Board of Directors in the US.

Maria Grazia Biferi unveiled the potential treatment strategy on April 25, 2017 at the ALS Association Drug Company Working Group, held during the 69th Annual Meeting of the American Academy of Neurology.

The approach builds on a previous strategy developed by Barkats team, using a related gene therapy delivery vehicle, for spinal muscular atrophy. A similar strategy, known as ChariSMA (AVXS-101), is now at the phase 1 stage and according to interim phase I results appears promising (see December 2015 news).

This is no longer an academic exercise, said Lucie Bruijn, Chief Scientist of the ALS Association. The current clinical trials in SMA confirm that this approach can succeed.

Going viral

In 2007, Martine Barkats team discovered that the recombinant adeno-associated virus AAV9 could penetrate the blood-brain barrier, opening the door to the development of gene therapies for motor neuron diseases. (see December 2008 news; Duque et al., 2009; Foust et al., 2009).

Every dog has its day. Researchers at Tufts University School of Medicine are now evaluating a potential gene therapy for SOD1-linked ALS in dogs. The approach aims to help dogs with a naturally occurring form of the disease known as canine degenerative myelopathy (DM), a late-onset disease first recognized in the 1970s in German Shepards. The trial is a key step toward developing a treatment for ALS, because dogs are larger and the disease is naturally occurring. [Image: Handicapped Pets. CC BY-SA 2.0 license.]

Building on these advances, research teams led by Nationwide Childrens Hospital Brian Kaspar in Ohio and University of Massachusetts Medical Centers Robert Brown began to develop potential therapies for SOD1 ALS. One of these strategies, being developed by Robert Brown and Christian Mueller at the University of Massachusetts Medical School, uses an artificial microRNA to reduce levels of mutant SOD1 synthesis in the brain and spinal cord. The approach is currently being evaluated at the preclinical stage in dogs with degenerative myelopathy (DM), a naturally occurring form of the disease. The clinical trial, being led by Tufts University School of Medicines Dominik Faissler in Massachusetts, launched in December 2016 and is ongoing.

The strategy builds in part, on previous studies led by Martine Barkats which found that AAV10 is more efficient than AAV9 in delivering genes into motor neurons in the spinal cord at lower doses, critical in developing a treatment for a disease (Tanguy et al., 2015). The approach, according to studies led by University of Californias Krystof S. Bankiewicz in San Francisco and University of Massachusetts Medical Centers Christian Mueller can be delivered efficiently intrathecally into motor neurons, at least in non-human primates.

Meanwhile, California Institute of Technologys Ben Deverman and Viviana Gradinaru used Cre-recombination-based AAV targeted evolution (CREATE), to develop novel gene therapy delivery vehicles that penetrate the blood brain barrier, to treat disorders of the central nervous system, including ALS. The vectors, which include AAV-PHP.B, can deliver genes into the motor cortex in the brain and the spinal cord at about 40 times greater efficiency compared to AAV9 upon intravenous injection (Deverman et al., 2016).

The delivery approach is now licensed to Cambridge startup Voyager Therapeutics in Massachusetts, which is also developing a gene therapy for SOD1 ALS. Voyager Therapeutics hopes to file an IND for their potential treatment strategy for ALS, known as VY-SOD101, at the end of 2017.

A key question is whether SOD1 is needed to reduce free radical levels that arise in key tissues affected by the disease. Therefore, a growing number of research teams are developing erase and replace strategies which aim to reduce mutant SOD1 while at the same time, produce the wild-type enzyme to help detoxify key tissues affected by ALS, including the brain, spinal cord and muscles.

A one, two punch?

Engineering a new approach. Researchers at University of Massachusetts Medical School are developing a CRISPR-Cas9-based gene therapy for SOD1-linked ALS that aims to permanently suppress mutant SOD1 synthesis. Meanwhile, Duke Universitys Charles Gersbach is developing a epigenomic editing-based strategy, also presented at AAN 2017, that may be of benefit to a wide range of diseases including the most common forms of ALS, C9orf72-linked disease (see January 2017 conference news; Thakore et al., 2015). [Courtesy of Ran et al., 2013, Nature Publishing Group.]

With the advent of new gene silencing technologies, research teams began to take another look at their gene therapy approaches and began to modify them in hopes to optimize their strategies to combat SOD1-linked disease. Robert Brown turned to CRISPR/Cas9 technologies in hopes to stop further mutant SOD1 synthesis. Their approach, unveiled at AAN 2017, uses imprecise CAS9-based editing to introduce indels in the SOD1 gene. The key obstacle, according to preliminary results presented by University of Massachusetts Medical Schools Zachary Taylor, is delivering sufficient Cas9 to modify the mutant SOD1 gene within motor neurons and glia in the central nervous system. Preclinical studies remain ongoing.

Across the globe, Martine Barkats and Maria Grazia Biferi, are developing a strategy using a novel gene silencing AAV10-based approach to tackle SOD1 ALS. The strategy is now being optimized and is at the preclinical stage. The approach involves the injection of the potential therapy into the blood and the brain. The reason, according to Biferi is to ensure that levels of the misfolded enzyme are reduced in key tissues outside the CNS including skeletal muscle.

This is important, explains Biferi, because ALS is beginning to be considered a multisystemic disease.

Now, the researchers are developing a similar strategy to tackle C9orf72 ALS, the most common form of the disease identified to date.

TARgeting ALS

A detox program for the CNS? Researchers at MeiraGTx in New York, in collaboration with Ronald Klein and Gregory Petsko, are developing a gene therapy strategy that aims to reduce the toxicity of TDP-43 by turning up nonsense-mediated mRNA decay. [Image: Emw, Wikimedia Commons.]

Meanwhile, Louisiana State Universitys Ronald Klein is setting his sights on helping to develop a gene therapy that targets more than 95% of cases of the disease. The approach, based on previous studies led by Gregory Petsko in New York, now at Weill Cornell Medical College in New York and Sami Barmada, now at the University of Michigan, aims to reduce cytoplasmic TDP43-mediated motor neuron toxicity by turning up nonsense-mediated mRNA decay. The gene therapy-based strategy increases levels of hUPF1, a key regulator of this process (see June 2015 news; Ju et al., 2011; Barmada et al., 2015).

The approach, according to a study from Kleins team, prolongs motor function at least for 8 weeks in a rat model of the disease. The study found that the potential therapy, when administered at day 1, preserved strength in the forelimbs of a rat model of ALS (see June 2015 news; Jackson et al., 2015).

It is really amazing that [increasing] hUPF has this really specific protective action against TDP-43, says Klein. We keep seeing it again and again.

How this strategy may mitigate TDP-43-mediated motor neuron toxicity remains unclear. The approach is one of at least two that aims to target TDP-43 buildup in the cytoplasm of motor neurons (see April 2017 news; Becker et al., 2017).

The strategy, now licensed to the New York biotech startup MeiraGTx, is at the preclinical stage. Evaluation of the approach in adult rat models of ALS is ongoing.

References

Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabreja GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH Jr, Mueller C. Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates. Hum Gene Ther. 2016 Jan;27(1):19-31. doi: 10.1089/hum.2015.122. [PubMed].

Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, Fyfe J, Moullier P, Colle MA, Barkats M. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther. 2009 Jul;17(7):1187-96. [PubMed].

Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol. 2009 Jan;27(1):59-65 [PubMed].

Tanguy Y, Biferi MG, Besse A, Astord S, Cohen-Tannoudji M, Marais T, Barkats M. Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice. Front Mol Neurosci. 2015 Jul 28;8:36. doi: 10.3389/fnmol.2015.00036. eCollection 2015. [PubMed].

Barmada SJ, Ju S, Arjun A, Batarse A, Archbold HC, Peisach D, Li X, Zhang Y, Tank EM, Qiu H, Huang EJ, Ringe D, Petsko GA, Finkbeiner S. Amelioration of toxicity in neuronal models of amyotrophic lateral sclerosis by hUPF1. Proc Natl Acad Sci U S A. 2015 Jun 23;112(25):7821-6. [PubMed].

Jackson KL, Dayton RD, Orchard EA, Ju S, Ringe D, Petsko GA, Maquat LE, Klein RL. Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis. Gene Ther. 2015 Jan;22(1):20-8. [PubMed].

Ju S, Tardiff DF, Han H, Divya K, Zhong Q, Maquat LE, Bosco DA, Hayward LJ, Brown RH Jr, Lindquist S, Ringe D, Petsko GA. A yeast model of FUS/TLS-dependent cytotoxicity. PLoS Biol. 2011 Apr;9(4):e1001052. [PubMed].

Becker LA, Huang B, Bieri G, Ma R, Knowles DA, Jafar-Nejad P, Messing J, Kim HJ, Soriano A, Auburger G, Pulst SM, Taylor JP, Rigo F, Gitler AD. Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice. Nature. 2017 Apr 20;544(7650):367-371. [PubMed].

Further Reading

van Zundert B, Brown RH Jr. Silencing strategies for therapy of SOD1-mediated ALS. Neurosci Lett. 2017 Jan 1;636:32-39. [PubMed].

Tora MS, Keifer OP Jr, Lamanna JJ, Boulis NM. The challenges of developing a gene therapy for amyotrophic lateral sclerosis. Expert Rev Neurother. 2017 Apr;17(4):323-325. [PubMed].

AAN2017 c9orf72 disease-als gene therapy SOD1 tdp-43 topic-preclinical topic-randd

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A New Potential ALS Gene Therapy Delivers A Key Milestone - ALS Research Forum

19 May 2017 Gene therapy might someday help mend badly broken bones This experimental method combines gene therapy, stem cells and ultrasound

Researchers have found that an experimental technique has healed large bone breaks in lab animals, researchers have found.

The technique is still far from becoming a reality, but, it repaired large bone gaps in the mini-pigs scientists studied. The hope, the researchers say, is to eventually help people with badly broken bones that won't heal.

The results of the study were published in Science Translational Medicine.

When a bone sustains a simple fracture, it is usually able to self-repair with time (and a cast). However, severe fractures can leave large gaps in the bone that the self-healing process cannot bridge.

The aim of gene therapy

Health24 previously reported that the basic function of genes is to regulate the production of proteins required for the healthy working of cells. Thus, genetic defects manifest in either too little or too much of a protein being produced.

The aim of gene therapy is to replace the defective gene with a healthy one. The correct amount of proteins will be produced, and the disease will then be cured.

Geneticists are literally snipping defective pieces out of a strand of DNA with molecular scissors called CRISPR.

Bone grafting

Right now, the "gold standard" treatment for those fractures is bone grafting, said Dan Gazit, one of the senior researchers on the new study.

There, surgeons take bone tissue from elsewhere in the body or from a donor and use it to repair the damaged bone.

When bone grafting is done with a patient's own tissue often taken from the pelvis that means additional surgery. And it can leave patients with prolonged pain and added risk of infection, Gazit said.

Newapproach

First, the researchers implanted a matrix of collagen a protein in bone into the gap between the two sides of a fractured bone.

That collagen then attracted the bone's resident stem cells, and gave them a structure to settle into. Stem cells are early cells that develop into mature tissue, including bone.

Once those cells have populated the gap in the bone, the next step involves gene therapy. The researchers injected a mixture of "microbubbles" and genetic material for a bone-promoting protein, called BMP, into the injury site.

That spurred the stem cells to form new bone tissue, according to the report.

In this study, the tactic healed bone breaks in all of the lab animals the researchers treated, Gazit said. In untreated pigs, the breaks did not heal, the findings showed.

A sophisticated approach needed

"But there are problems with BMP," said Dr Joseph Lane, an orthopaedic trauma surgeon at the Hospital for Special Surgery in New York City.

A central issue, he said, is that very high BMP doses may be needed, and side effects including infections and excess bone growth are common.

The new approach is "going in that direction", said Lane, who was not involved in the research. The "beauty" of it, he said, is that it harnesses the bone-promoting effects of BMP in a more natural way.

A number of questions should be answered before human trials are done, according to Lane. For example, he said, future animal research should look at more complicated fractures. The bone injuries used in this study are relatively easy to heal, versus severe fractures, Lane said.

Read more:

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Menopause before 40 carries higher risk of broken bones

Patients in Mpumalanga are going home with broken bones

People with recessive dystrophic epidermolysis bullosa aren't able to produce a protein that binds the upper and lower levels of skin together, and at the slightest friction, these layers slide and create blisters.

Tereza is the CEO of the National Osteoporosis Foundation and worked as a Nursing Sister in the field of Osteoporosis for 18 years prior to her appointment with the Foundation. She used to be the Educational Officer for the Foundation and co-wrote the patient brochure on Osteoporosis. Read more

The information provided does not constitute a diagnosis of your condition. You should consult a medical practitioner or other appropriate health care professional for a physical exmanication, diagnosis and formal advice. Health24 and the expert accept no responsibility or liability for any damage or personal harm you may suffer resulting from making use of this content.

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Gene therapy might someday help mend badly broken bones ... - Health24

In Brief Researchers from Johns Hopkins Medicine in Maryland have discovered a rather unusual way to treat a severe form of age-related blindness. They found a virus inserted into the retina can be used to halt or even reverse the disease. A Unique Treatment

They say you dont fight fire with fire. However, researchers from Johns Hopkins Medicine in Maryland have found that sometimes a virus may be the best weapon against a disease.Their studyhas been publishedin The Lancet

The researchers werelooking for ways to treat a particular type ofage-related macular degeneration (AMD)known as a wet AMD. Its a rare and more severe form of the disease,affecting just 10 percent of all AMD patients, and it causes new blood vessels to grow under the retina, which then leak blood and fluid into the eye, leading to vision problems.

The researchers knewthey could halt and even reverse the condition by suppressing an overactive protein called vascular endothelial growth factor (VEGF). Other researchers had been able to do it with monthly eye injections, but this team was hoping to do it with just one injection.

The best way they found to do this was by using a common cold-like virus called AAV2 as a carrier of gene that activates the production of a differentprotein,sFLT01, tocounter VEGF.

In a preliminary trial involving 19 men and women 50 years old and above, the researchers injected the patients with a form of AAV2that was genetically engineered to penetrate retinal cells and deposit the gene. After the virus deposited the gene, the cells began secreting sFLT01 which bound to VEGF and prevented it from stimulating leakage and growth of abnormal blood vessels, explained a Johns Hopkins press release.

The clinical trial showed promising results, with the condition of four of the patients improving dramatically after just one viral injection. Two others saw some reduction in the fluid build up, and the treatment didnt produce any side effects in any patients. Even at the highest dose, the treatment was quite safe. We found there were almost no adverse reactions in our patients, said researcher Peter Campochiaro.

Of the patients that didnt respond, the researchers discovered that five naturally produced antibodies that would attack the AAV2 virus, rendering it unable to complete its gene depositing mission. They think these antibodies could be prevalent throughout the population, making it difficult to determine how effecting the treatment would actually be.

Nevertheless, this research is a step in the right direction, especially with AMD expected to affect almost 5.44 million people in the U.S. by 2050. This preliminary study is a small but promising step towards a new approach that will not only reduce doctor visits and the anxiety and discomfort associated with repeated injections in the eye, but may improve long-term outcomes, Campochiaro said.

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An Experimental Gene Therapy Uses Viruses to Stop Age-Related ... - Futurism

New gene therapy for vision loss is safe in humans, study suggests Science Daily In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving ... and more »

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New gene therapy for vision loss is safe in humans, study suggests - Science Daily

By Robert F. ServiceMay. 17, 2017 , 2:30 PM

It takes more than a cast and a little time to heal many broken bones. Whether its a soldier wounded in battle, a car accident victim, or an elderly person who has fallen, bone damage can be so extensive that the bones never heal properly, leaving people crippled or with other severe problems. Now, researchers have combined ultrasound, stem cells, and gene therapy to stimulate robust bone repair. So far the work has only been performed in animals. But it has already been so successful that its expected to move quickly toward human clinical trials.

The new research has huge clinical significance, says David Kulber, who directs the Center for Plastic and Reconstructive Surgery at Cedars-Sinai Medical Center in Los Angeles, California, and who was not part of the study. The technology of being able to stimulate bone growth is really remarkable.

Its also one for which there is a glaring need. In the United States alone, some 100,000 people a year suffer from what is known as a nonunion fracture. In these cases, parts of a bone may be missing altogether or so badly splintered that the bone cant be reassembled. In such cases, doctors typically graft other bone into the site. Ideally this bone comes from the same personoften taken from the pelvis, a painful procedure that compounds a persons injuries and recovery time. When this isnt possible, physicians will turn to cadavers for the extra bone. But this bone must be sterilized before its implanted, robbing it of proteins and other signaling molecules that encourage its regrowth once transplanted, and lessening the chances of a full recovery.

Researchers have long tried to improve matters by growing new bone without use of a graft. To do so they typically first fill gaps in bone with a natural scaffolding material called collagen. This scaffolding encourages a persons own bone-forming stem cells, called mesenchymal stem cells (MSCs), to migrate into the area. The trouble is MSCs dont only differentiate into osteocytes, the bone-producing cells. They can also develop into either fat tissue cells or scar tissue.

Researchers have tried for years to steer MSCs into becoming osteocytes by exposing them to one or more bone morphogenetic proteins (BMPs), signaling molecules that trigger the cells to transform into bone-forming cells. But for this differentiation to occur, MSCs must be exposed to BMPs for up to a week. Yet if the BMPs are simply injected into the site of a fracture, they dissipate in just hours.

In an effort to produce a lasting BMP signal, researchers led by Dan Gazit, a regenerative medicine expert at Cedars-Sinai, as well as other groups, have previously turned to using viruses to introduce extra copies of BMP genes into MSCs so that the cells themselves will produce the proteins long enough to trigger their own differentiation. But success has been halting here, too.

Over the last several years, Gazits teamamong othershasdeveloped an alternative strategy for efficiently getting genes into MSCs without viruses. The researchers start by packing the wound with the usual collagen matrix and waiting for a couple of weeks for the stem cells to infiltrate the scaffold. They then create a solution containing numerous copies of their gene of interest alongside gas-filled micron-sized bubbles encased by a thin shell of fat molecules. After injecting this solution into the fracture site, they go over the area with an ultrasound wand, much as its done by obstetricians to check on the health of a fetus. The wands ultrasound pulses burst the microbubbles, briefly punching nano-sized holes in any adjacent stem cells, which allows the genes in the solution to enter.

In 2014, Gazit and his colleagues reported that they used this procedure to introduce nontherapeutic reporter genes into large fractures in animal models. But when they used the procedure to introduce genes for two different BMPsBMP-2 and BMP-7they detected some bone regrowth in the animals, but not enough to heal the fractures.

Gazits group has gotten better results by using the same approach to insert copies of the gene for BMP-6 into pigs that had been surgically given 1-centimeter gaps in a leg bone. After waiting 8 weeks, they found that the bone gap was closed and the leg fracture was healed in all of the treated animals. In fact, the procedure was so effective that the fractures healed as well as when bone grafts were carried out using bone from the same animal, the currently preferred treatment, they report today in Science Translational Medicine.

The results are just the type of thing we need to move this field forward, says Johnny Huard, an orthopedics researcher at the University of Texas Health Science Center in Houston. However, he notes, the pigs used in this study were all under 1 year in age. Younger animals, including people, tend to have far more MSCs than older ones, he says, yet large fractures are far more common in the elderly than the young. So Huard suggests that before the approach is ready for testing in people with bone fractures, it would be good to first see whether its equally successful in older animals.

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Tiny bubbles and a bit of gene therapy heal major bone fractures in pigs - Science Magazine

SB-525 has already received Orphan Drug designation from the FDA. The FDA has cleared an Investigational New Drug application for this program, and a Phase 1/2 clinical trial evaluating SB-525 in adults with hemophilia A is expected to open and begin screening subjects for enrollment by the end of the second quarter 2017. Data from this study are expected in late 2017 or early 2018.

About Hemophilia A

Hemophilia A is a monogenic, rare bleeding disorder in which the blood does not clot normally. It is caused by mutations in the F8 gene which encodes Factor VIII clotting protein that helps the blood clot and stop bleeding when blood vessels are injured. Individuals with this mutation experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. According to the Centers for Disease Control and Prevention, hemophilia occurs in about one of every 5,000 male births, with an estimated 20,000 males in the U.S. living with the disorder.

About Sangamo Therapeutics

Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at http://www.sangamo.com.

Forward Looking Statements

Thispressreleasemaycontainforward-looking statements based on Sangamo's current expectations. Theseforward-looking statements include, without limitation references relating to the benefit of Fast Track designation to accelerate regulatory approval of SB-525, research and development of therapeutic applications of Sangamo's gene therapy and ZFP technology platforms, the potential of Sangamo's technology to treat hemophilia and lysosomal storage disorders, and the expected timing of initiating clinical trials of SB-525 and the release of data from these trials. Actual results may differ materially from these forward-looking statements due to a number of factors, includinguncertaintiesrelatingto substantial dependence on the clinical success of lead therapeutic programs,the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's SEC filings, including the risk factors described in its Annual Report onForm10-K and its most recent QuarterlyReportonForm10-Q. Sangamo Therapeutics, Inc. assumes no obligation to update the forward-looking information contained in this pressrelease.

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Sangamo Receives Fast Track Designation From The FDA For SB-525 Investigational Hemophilia A Gene Therapy - PR Newswire (press release)

MOUNTAIN VIEW, CA--(Marketwired - May 15, 2017) - SENS Research Foundation (SRF) has launched a new research program focused on somatic gene therapy in collaboration with the Buck Institute for Research on Aging. Brian Kennedy, PhD, a leading expert on the biology of aging, will be running the project in his lab at the Buck.

Many potential treatments of age-related diseases require the addition of new genes to the genome of cells in the body, a technology known as somatic gene therapy. The technology has been hampered, up until now, by the inability to control where the gene is inserted. That lack of control resulted in a significant risk of insertion in a location that encourages the cell to become malignant.

SRF has devised a new method for inserting genes into a pre-defined location. In this program, this will be done as a two-step process, in which first CRISPR is used to create a "landing pad" for the gene, and then the gene is inserted using an enzyme that only recognizes the landing pad. SRF has created "maximally modifiable mice" that already have the landing pad, and this project will evaluate how well the insertion step works in different tissues.

"Somatic gene therapy has been a goal of medicine for decades. Being able to add new healthy genes will enable us to address treatments of such age-related diseases as atherosclerosis and macular degeneration. Our collaboration with SRF will substantially move us toward finding effective treatments to genetically based age-related diseases," said Dr. Kennedy.

"Partnering with Brian Kennedy and the Buck enables SRF to continue towards our goal of achieving human clinical trials on rejuvenation biotechnologies in the next five years. Brian's leadership in moving this technology into mammals is a huge step forward," said Dr. Aubrey de Grey, CSO, SENS Research Foundation.

This research has been made possible through the generous support of the Forever Healthy Foundation and its founder Michael Greve, as well as the support of our other donors. The Forever Healthy Foundation is a private nonprofit initiative whose mission is to enable people to vastly extend their healthy lifespans and be part of the first generation to cure aging. In order to accelerate the development of therapies to bring aging under full medical control, the Forever Healthy Foundation directly supports cutting-edge research aimed at the molecular and cellular repair of damage caused by the aging process.

About SENS Research Foundation (SRF)SENS Research Foundation is a 501(c)(3) nonprofit that works to research, develop, and promote comprehensive regenerative medicine solutions for the diseases of aging. SRF is focused on a damage repair paradigm for treating the diseases of aging, which it advances through scientific research, advocacy, and education. SENS Research Foundation supports research projects at universities and institutes around the world with the goal of curing such age-related diseases as macular degeneration, heart disease, cancer, and Alzheimer's disease. Educating the public and training researchers to support a growing regenerative medicine field are also major endeavors of the organization that are being accomplished though advocacy campaigns and educational programs. For more information, visit http://www.sens.org.

About Buck Institute for Research on AgingBuck Institute is the U.S.'s first independent research organization devoted to Geroscience -- focused on the connection between normal aging and chronic disease. Based in Novato, California, the Buck is dedicated to extending "healthspan," the healthy years of human life, and does so by utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and others focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimer's and Parkinson's, cancer, cardiovascular disease, macular degeneration, osteoporosis, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics, bioinformatics and stem cell technologies. For more information: http://www.thebuck.org.

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SENS Research Foundation Announces New Research Program on Somatic Gene Therapy With Buck Institute for ... - Markets Insider

A gene therapy that deliberately infects the eye with a virus can safely preserve vision in people affected by one of the leading causes of blindness, research has shown.

In a small preliminary study, scientists used an altered common cold-type virus to carry a repair gene that combats age-related macular degeneration (AMD).

The disease is marked by abnormal blood vessels that leak fluid into the central part of the retina, or macula.

After being injected into patients' eyes, the virus penetrated retinal cells and deposited the gene, which manufactured a therapeutic protein called FLT01.

Lead researcher Professor Peter Campochiaro, from Johns Hopkins University in the US, said: This preliminary study is a small but promising step towards a new approach that will not only reduce doctor visits and the anxiety and discomfort associated with repeated injections in the eye, but may improve long-term outcomes.

The Phase I clinical trial involved 19 men and women aged 50 and older with advanced wet AMD.

With the help of the gene, retinal cells were turned into factories making FLT01.

The scientists hope this will eliminate the need to administer repeated injections of the protein, which suppresses a natural growth-driving molecule called VEGF.

Prolonged suppression of VEGF is needed to preserve vision, and that is difficult to achieve with repeated injections because life often gets in the way, said Prof Campochiaro.

For safety and ethical reasons, the patient group consisted of people for whom standard approved treatments were highly unlikely to restore vision.

Only 11 patients stood any chance of fluid reduction. Of those, four showed dramatic improvements after the gene therapy. The amount of fluid in their eyes dropped from a severe level to almost nothing.

Two other patients experienced a partial reduction in the amount of fluid in their eyes.

The findings are reported in the latest issue of The Lancet medical journal.

Press Association

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Gene therapy infection can prevent blindness, research shows - The Independent

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Sangamo Therapeutics, Inc. (NASDAQ: SGMO) today highlighted data from research and clinical-stage programs presented over the past week at the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT). Research from Sangamo scientists and collaborators was selected for 10 oral presentations and nine poster presentations during the conference.

"This year at ASGCT we showcased several exciting research and clinical programs emerging from Sangamo's laboratories," said Dr. Sandy Macrae, Sangamo's CEO. "Sangamo is known for its leading research in genome editing, and over time we have developed additional expertise in gene therapy, gene regulation and cell therapy. We are also rapidly advancing our viral and non-viral delivery capabilities which have the potential to broaden our applications of genomic therapies. Such range of expertise allows us to be selective as we pair technology platforms with therapeutic applications, and compels us to make strategic choices about our product candidates. We will develop and commercialize certain products ourselves, while others, such as our gene therapy for hemophilia A now in collaboration with Pfizer, or our CNS or oncology programs, we may advance with a partner to leverage the right disease area focus, skills and resources."

Selected Highlights from ASGCT 2017

Zinc Finger Nuclease Technology ImprovementsEd Rebar, Ph.D., Sangamo's vice president of technology, presented recent enhancements to the Company's zinc finger nuclease (ZFN) genome editing technology that substantially improve specificity while maintaining very high levels of on-target modification. These include the removal of positively charged amino acids in the zinc finger beta-sheet that make non-specific contacts with the DNA phosphate backbone, as well as the substitution of key residues within the Fok-1 cleavage domain. Dr. Rebar showed that these refinements could be applied broadly to ZFN reagents to substantially reduce off-target cleavage without sacrificing on-target cutting efficiency.

Dr. Rebar concluded with a detailed specificity analysis of a ZFN pair, in which these approaches were combined, which identified no significant off-target modification with an assay sensitivity of approximately 0.1%. Importantly, this study was performed on samples generated using clinically relevant delivery conditions, transfection scales and cell types, and with an on-target modification level of greater than 80%.

Gene Therapy for Fabry DiseaseThomas Wechsler, Ph.D., Sangamo's director and lead scientist for rare diseases, presented new data from the Company's preclinical AAV-cDNA gene therapy program for Fabry disease. Earlier in the week, Sangamo announced that it will advance this program toward human clinical development with preclinical studies enabling an Investigational New Drug Application (IND) in the second half 2018.

Fabry is an X-linked lysosomal storage disorder caused by mutations in the GLA gene that encodes for the alpha-galactosidase A enzyme (-Gal A). This mutation results in the buildup of Gb3 and Lyso-Gb3 lipid molecules in the body's cells, resulting in a range of symptoms and life-threatening complications that affect multiple tissues and organ systems in the body.

Dr. Weschler presented data from GLAKO mouse models of Fabry disease demonstrating that a single infusion of Sangamo's AAV vector containing an -Gal A transgene and a liver specific promoter successfully transduced the liver, resulting in episomal expression of -Gal A in the plasma and various tissues for the duration of the study, out to 60 days. From a single treatment, the AAV-cDNA vector achieved enzyme activity levels in the plasma of up to 100 fold greater than wildtype and 10 to 100 fold greater than wildtype in tissues including the liver, heart, kidney and spleen. Importantly, -Gal A secreted from the liver led to a significant reduction in the levels of accumulated Gb3 and Lyso-Gb3 lipid substrates, in target tissues such as the kidney and heart.

Gene Regulation Treatment for Reduction of TauSangamo Scientist Bryan Zeitler, Ph.D., presented recent data demonstrating significant reduction of tau expression using Sangamo's proprietary zinc finger protein transcription factor (ZFP-TF) gene-regulation technology. The research was conducted in conjunction with Dr. Brad Hyman, Director of the Alzheimer's Disease Research Center at Massachusetts General Hospital. The reduction of tau expression has been shown to help reduce neurofibrillary tangles in the brain and provide neuronal protection and reversal of pathology in Alzheimer's disease and other tauopathy disease models.

The presentation included data from in vivo studies in wild-type mice demonstrating up to 90% reduction of tau mRNA and protein in the mouse hippocampus, as well as up to 70% tau reduction across all regions of the brain, including the cortex, midbrain, cerebellum, thalamus, hypothalamus and striatum.

In addition, data from in vivo studies in an amyloid mouse model of Alzheimer's disease suggest that a single administration of ZFP-TFs significantly reduced neuronal dystrophies in mice with established disease pathology. This is the first time that a tau lowering agent has demonstrated a reduction in neuritic dystrophy. Specificity and off-target analysis in ZFP-TF-treated primary neurons revealed that tau was the only gene suppressed out of more than 26,000 coding transcripts analyzed. New data in Dr. Zeitler's presentation demonstrated that the effect of ZFP-TF treatment in lowering tau was durable out to the last measurement, at 11 months.

These experiments were conducted using Sangamo's novel, proprietary AAV serotype for improved CNS transduction.

Sangamo intends to seek a partner with disease area expertise for the development and commercialization of its gene regulation approach for certain central nervous system applications including Alzheimer's disease and other tauopathies.

In Vivo Genome Editing Treatments for MPS I and MPS IISangamo Scientist Russell DeKelver, Ph.D., presented additional preclinical data from the Company's in vivo genome editing clinical programs in MPS I and MPS II demonstrating phenotypic correction of disease in mouse models following a single administration of Sangamo's genome editing treatments. Newly presented histopathological analysis demonstrated reduced cellular vacuolation in various secondary tissues, as well as in the bone marrow, and central nervous system tissues such as the spinal cord and pituitary gland in treated MPS I and MPS II mice, four months after dosing. Furthermore, newly presented mass spectrometry analysis confirmed significant reduction of dermatan sulfate, a type of GAG biomarker, in the brains of MPS I and MPS II mice treated with Sangamo's genome editing treatments.

Sangamo recently initiated two Phase 1/2 clinical trials evaluating SB-318 and SB-913, ZFN-mediated in vivo genome editing treatments for MPS I and MPS II, respectively. Data are expected in late 2017 or early 2018.

Cell TherapyResearch by Brigit Riley, Ph.D., Sangamo's director of discovery and translational research, was presented demonstrating high levels of homology driven genome editing of human B cells by ZFN mRNA and AAV6 transgene delivery. The data demonstrated robust ZFN-mediated, site-specific modification of B cells at targeted loci, including AAVS1, CCR5 and TRAC locus. The data also demonstrated high levels of targeted transgene insertion, driven by homology directed repair, using a B cell specific promoter. Analysis of AAV serotype transduction showed the superiority of AAV6 in transducing B cells compared to several other serotypes.

The data demonstrate the potential for using genome editing to genetically modify B cells ex vivo and harness their natural ability to produce large amounts of antibodies to generate protein production reservoirs. This novel approach for using genome editing to harness the protein production capacity of B cells could be relevant for multiple indications, including immune disorders, cancer immunotherapies and other monogenic disorders.

DeliverySangamo Scientist Anthony Conway, Ph.D., presented new data from the Company's research into a next-generation delivery platform using lipid nanoparticles (LNPs). ZFN mRNA delivery via LNPs allowed for accumulation of genome modification within the mouse liver following repeat administration, with progressive increases in genomic modification out to six repeat doses tested. LNP delivery of new ZFN architectures led to greater than 85% on-target modification in vitro and greater than 60% on-target modification in vivo, resulting in greater than 90% protein knockdown of TTR and PCSK9 in wildtype mice. Repeat dosing of ZFNs using LNP-mRNA in combination with a single human AAV-IDS donor vector resulted in efficient targeted insertion of the IDS gene into the albumin locus and accumulative enzymatic activity levels in mouse plasma after each subsequent dose.

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Sangamo Biosciences (SGMO) Presents Recent Developments from Research and Clinical Programs at ASGCT - StreetInsider.com

Selected Highlights from ASGCT 2017

Zinc Finger Nuclease Technology ImprovementsEd Rebar, Ph.D., Sangamo's vice president of technology, presented recent enhancements to the Company's zinc finger nuclease (ZFN) genome editing technology that substantially improve specificity while maintaining very high levels of on-target modification. These include the removal of positively charged amino acids in the zinc finger beta-sheet that make non-specific contacts with the DNA phosphate backbone, as well as the substitution of key residues within the Fok-1 cleavage domain. Dr. Rebar showed that these refinements could be applied broadly to ZFN reagents to substantially reduce off-target cleavage without sacrificing on-target cutting efficiency.

Dr. Rebar concluded with a detailed specificity analysis of a ZFN pair, in which these approaches were combined, which identified no significant off-target modification with an assay sensitivity of approximately 0.1%. Importantly, this study was performed on samples generated using clinically relevant delivery conditions, transfection scales and cell types, and with an on-target modification level of greater than 80%.

Gene Therapy for Fabry DiseaseThomas Wechsler, Ph.D., Sangamo's director and lead scientist for rare diseases, presented new data from the Company's preclinical AAV-cDNA gene therapy program for Fabry disease. Earlier in the week, Sangamo announced that it will advance this program toward human clinical development with preclinical studies enabling an Investigational New Drug Application (IND) in the second half 2018.

Fabry is an X-linked lysosomal storage disorder caused by mutations in the GLA gene that encodes for the alpha-galactosidase A enzyme (-Gal A). This mutation results in the buildup of Gb3 and Lyso-Gb3 lipid molecules in the body's cells, resulting in a range of symptoms and life-threatening complications that affect multiple tissues and organ systems in the body.

Dr. Weschler presented data from GLAKO mouse models of Fabry disease demonstrating that a single infusion of Sangamo's AAV vector containing an -Gal A transgene and a liver specific promoter successfully transduced the liver, resulting in episomal expression of -Gal A in the plasma and various tissues for the duration of the study, out to 60 days. From a single treatment, the AAV-cDNA vector achieved enzyme activity levels in the plasma of up to 100 fold greater than wildtype and 10 to 100 fold greater than wildtype in tissues including the liver, heart, kidney and spleen. Importantly, -Gal A secreted from the liver led to a significant reduction in the levels of accumulated Gb3 and Lyso-Gb3 lipid substrates, in target tissues such as the kidney and heart.

Gene Regulation Treatment for Reduction of TauSangamo Scientist Bryan Zeitler, Ph.D., presented recent data demonstrating significant reduction of tau expression using Sangamo's proprietary zinc finger protein transcription factor (ZFP-TF) gene-regulation technology. The research was conducted in conjunction with Dr. Brad Hyman, Director of the Alzheimer's Disease Research Center at Massachusetts General Hospital. The reduction of tau expression has been shown to help reduce neurofibrillary tangles in the brain and provide neuronal protection and reversal of pathology in Alzheimer's disease and other tauopathy disease models.

The presentation included data from in vivo studies in wild-type mice demonstrating up to 90% reduction of tau mRNA and protein in the mouse hippocampus, as well as up to 70% tau reduction across all regions of the brain, including the cortex, midbrain, cerebellum, thalamus, hypothalamus and striatum.

In addition, data from in vivo studies in an amyloid mouse model of Alzheimer's disease suggest that a single administration of ZFP-TFs significantly reduced neuronal dystrophies in mice with established disease pathology. This is the first time that a tau lowering agent has demonstrated a reduction in neuritic dystrophy. Specificity and off-target analysis in ZFP-TF-treated primary neurons revealed that tauwas the only gene suppressed out of more than 26,000 coding transcripts analyzed. New data in Dr. Zeitler's presentation demonstrated that the effect of ZFP-TF treatment in lowering tau was durable out to the last measurement, at 11 months.

These experiments were conducted using Sangamo's novel, proprietary AAV serotype for improved CNS transduction.

Sangamo intends to seek a partner with disease area expertise for the development and commercialization of its gene regulation approach for certain central nervous system applications including Alzheimer's disease and other tauopathies.

In Vivo Genome Editing Treatments for MPS I and MPS IISangamo Scientist Russell DeKelver, Ph.D., presented additional preclinical data from the Company's in vivo genome editing clinical programs in MPS I and MPS II demonstrating phenotypic correction of disease in mouse models following a single administration of Sangamo's genome editing treatments. Newly presented histopathological analysis demonstrated reduced cellular vacuolation in various secondary tissues, as well as in the bone marrow, and central nervous system tissues such as the spinal cord and pituitary gland in treated MPS I and MPS II mice, four months after dosing. Furthermore, newly presented mass spectrometry analysis confirmed significant reduction of dermatan sulfate, a type of GAG biomarker, in the brains of MPS I and MPS II mice treated with Sangamo's genome editing treatments.

Sangamo recently initiated two Phase 1/2 clinical trials evaluating SB-318 and SB-913, ZFN-mediated in vivo genome editing treatments for MPS I and MPS II, respectively. Data are expected in late 2017 or early 2018.

Cell TherapyResearch by Brigit Riley, Ph.D.,Sangamo's director of discovery and translational research, was presented demonstrating high levels of homology driven genome editing of human B cells by ZFN mRNA and AAV6 transgene delivery. The data demonstrated robust ZFN-mediated, site-specific modification of B cells at targeted loci, including AAVS1, CCR5 and TRAC locus. The data also demonstrated high levels of targeted transgene insertion, driven by homology directed repair, using a B cell specific promoter. Analysis of AAV serotype transduction showed the superiority of AAV6 in transducing B cells compared to several other serotypes.

The data demonstrate the potential for using genome editing to genetically modify B cells ex vivo and harness their natural ability to produce large amounts of antibodies to generate protein production reservoirs. This novel approach for using genome editing to harness the protein production capacity of B cells could be relevant for multiple indications, including immune disorders, cancer immunotherapies and other monogenic disorders.

DeliverySangamo Scientist Anthony Conway, Ph.D., presented new data from the Company's research into a next-generation delivery platform using lipid nanoparticles (LNPs). ZFN mRNA delivery via LNPs allowed for accumulation of genome modification within the mouse liver following repeat administration, with progressive increases in genomic modification out to six repeat doses tested. LNP delivery of new ZFN architectures led to greater than 85% on-target modification in vitro and greater than 60% on-target modification in vivo, resulting in greater than 90% protein knockdown of TTR and PCSK9 in wildtype mice. Repeat dosing of ZFNs using LNP-mRNA in combination with a single human AAV-IDS donor vector resulted in efficient targeted insertion of the IDS gene into the albumin locus and accumulative enzymatic activity levels in mouse plasma after each subsequent dose.

About SangamoSangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has a strategic collaboration with Pfizer, Inc. for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at http://www.sangamo.com.

Forward Looking StatementsThis press release contains forward-looking statements regarding Sangamo's current expectations. These forward looking statements include, without limitation, references to the potential of novel delivery systems to broaden applications of genomic therapies,the ability to bring research and preclinical studies to clinical development, the expected timing of filing INDs and releasing data from ongoing clinical programs, the intent to seek partners and collaborators to develop and commercialize gene regulation treatment, and the research and development of ZFNs and ZFP-TFs, clinical trials and therapeutic applications of Sangamo's ZFP technology. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo's therapeutics, and the ability to establish strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable gene-based therapeutics. 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 Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q as filed with the Securities and Exchange Commission. 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.

ContactSangamo Therapeutics, Inc. McDavid Stilwell (510) 970-6000, x219 mstilwell@sangamo.com

Varant Shirvanian (510) 970-6000, x205 vshirvanian@sangamo.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-presents-recent-developments-from-research-and-clinical-programs-at-annual-meeting-of-the-american-society-of-gene--cell-therapy-300457323.html

SOURCE Sangamo Therapeutics, Inc.

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Sangamo Therapeutics Presents Recent Developments from Research and Clinical Programs at Annual Meeting of the ... - PR Newswire (press release)

MOUNTAIN VIEW, CA--(Marketwired - May 15, 2017) - SENS Research Foundation (SRF) has launched a new research program focused on somatic gene therapy in collaboration with the Buck Institute for Research on Aging. Brian Kennedy, PhD, a leading expert on the biology of aging, will be running the project in his lab at the Buck.

Many potential treatments of age-related diseases require the addition of new genes to the genome of cells in the body, a technology known as somatic gene therapy. The technology has been hampered, up until now, by the inability to control where the gene is inserted. That lack of control resulted in a significant risk of insertion in a location that encourages the cell to become malignant.

SRF has devised a new method for inserting genes into a pre-defined location. In this program, this will be done as a two-step process, in which first CRISPR is used to create a "landing pad" for the gene, and then the gene is inserted using an enzyme that only recognizes the landing pad. SRF has created "maximally modifiable mice" that already have the landing pad, and this project will evaluate how well the insertion step works in different tissues.

"Somatic gene therapy has been a goal of medicine for decades. Being able to add new healthy genes will enable us to address treatments of such age-related diseases as atherosclerosis and macular degeneration. Our collaboration with SRF will substantially move us toward finding effective treatments to genetically based age-related diseases," said Dr. Kennedy.

"Partnering with Brian Kennedy and the Buck enables SRF to continue towards our goal of achieving human clinical trials on rejuvenation biotechnologies in the next five years. Brian's leadership in moving this technology into mammals is a huge step forward," said Dr. Aubrey de Grey, CSO, SENS Research Foundation.

This research has been made possible through the generous support of the Forever Healthy Foundation and its founder Michael Greve, as well as the support of our other donors. The Forever Healthy Foundation is a private nonprofit initiative whose mission is to enable people to vastly extend their healthy lifespans and be part of the first generation to cure aging. In order to accelerate the development of therapies to bring aging under full medical control, the Forever Healthy Foundation directly supports cutting-edge research aimed at the molecular and cellular repair of damage caused by the aging process.

About SENS Research Foundation (SRF)SENS Research Foundation is a 501(c)(3) nonprofit that works to research, develop, and promote comprehensive regenerative medicine solutions for the diseases of aging. SRF is focused on a damage repair paradigm for treating the diseases of aging, which it advances through scientific research, advocacy, and education. SENS Research Foundation supports research projects at universities and institutes around the world with the goal of curing such age-related diseases as macular degeneration, heart disease, cancer, and Alzheimer's disease. Educating the public and training researchers to support a growing regenerative medicine field are also major endeavors of the organization that are being accomplished though advocacy campaigns and educational programs. For more information, visit http://www.sens.org.

About Buck Institute for Research on AgingBuck Institute is the U.S.'s first independent research organization devoted to Geroscience -- focused on the connection between normal aging and chronic disease. Based in Novato, California, the Buck is dedicated to extending "healthspan," the healthy years of human life, and does so by utilizing a unique interdisciplinary approach involving laboratories studying the mechanisms of aging and others focused on specific diseases. Buck scientists strive to discover new ways of detecting, preventing and treating age-related diseases such as Alzheimer's and Parkinson's, cancer, cardiovascular disease, macular degeneration, osteoporosis, diabetes and stroke. In their collaborative research, they are supported by the most recent developments in genomics, proteomics, bioinformatics and stem cell technologies. For more information: http://www.thebuck.org.

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SENS Research Foundation Announces New Research Program on ... - Marketwired (press release)

May 15, 2017 08:00 ET | Source: Selecta Biosciences

WATERTOWN, Mass., May 15, 2017 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ:SELB), a clinical-stage biopharmaceutical company focused on unlocking the full potential of biologic therapies by avoiding unwanted immune responses, today announced new preclinical data regarding non-immunogenic gene therapies that were presented at the American Society of Gene & Cell Therapy (ASGCT) 2017 Annual Meeting, which took place last week in Washington, D.C.

Immunogenicity is a key challenge in gene therapy, limiting the number of diseases and patients that can be effectively treated and presenting a safety hurdle, said Werner Cautreels, Ph.D., CEO and Chairman of Selecta. Together with various collaborators, we have again demonstrated the potential of Selectas proprietary immune tolerance Synthetic Vaccine Particles (SVP) technology, which is designed to improve the clinical benefits and transform the development of gene therapy. We also were pleased with the presentation of preclinical proof-of-concept data for our proprietary product candidate in MMA, a life-threatening rare disease that can only be treated today by diet or organ transplantation.

A team led by Charles Venditti, M.D., Ph.D., Senior Investigator and Head, Organic Acid Research Section in the National Human Genome Research Institute at the National Institutes of Health, and Luk Vandenberghe, Ph.D., Director of the Grousbeck Gene Therapy Center at Mass. Eye and Ear and an Assistant Professor at Harvard Medical School, delivered a presentation entitled Anc80 Mediates Hepatic Correction of Methylmalonyl-CoA Mutase Deficiency in Murine Models of Methymalonic Acidemia. This presentation featured data from mouse models of MMA, a rare inborn error of metabolism most frequently caused by mutations in the enzyme methylmalonyl-CoA mutase (MUT). In this study, MUT-deficient mice were treated with Selectas Anc80-synMUT product candidate to express the human MUT gene. The gene therapy induced a robust biochemical and clinical response as plasma methylmalonic acid levels dropped precipitously, substantial weight gain ensued and survival was sustained. Further, presented data indicate that the combination of SVP and Anc80 could effectively overcome the immunogenicity that has limited other gene therapy programs by enabling enrollment of patients with pre-existing antibodies to AAV and keeping patients eligible for repeat administration.

A team led by Federico Mingozzi, Ph.D., Head of Immunology and Liver Gene Therapy at Genethon, delivered a presentation entitled Modulation of AAV Vector Dosing and Avoidance of Capsid Immune Responses via Repeated Co-Administration of Vector with Rapamycin Tolerogenic Nanoparticles. This presentation featured data from both mouse and non-human primate studies demonstrating how the co-administration of SVP-Rapamycin completely blocked anti-AAV immune responses in an antigen-specific manner and allowed for vector re-administration and gene therapy dose titration. The ability to dose titrate could provide for more effective development and administration of gene therapies.

Click here to view these presentations.

About Selecta's MMA Program

MMA is an inborn error of metabolism that, according to the U.S. National Institutes of Health (NIH), affects an estimated one in 25,000 to 48,000 individuals globally. MMA patients are unable to process certain proteins and fats, leading to the accumulation of toxic metabolites. Symptoms of this life-threatening disease start to develop in early childhood and, despite strict diet, patients suffer from a wide range of disease-related complications such as pancreatitis, strokes and chronic kidney failure. Selecta exclusively licensed Anc80 for MMA from Massachusetts Eye and Ear (MEE) in May 2016. Under the license agreement, Selecta also has the exclusive option to develop gene therapies using Anc80 for additional pre-defined lysosomal storage, genetic muscular and genetic metabolic diseases. In early 2017, Selecta entered into a strategic manufacturing agreement with Lonza Houston, Inc. under which Lonzawill produce an Anc80-AAV-based gene therapy product for Selecta's MMA program.

Selecta intends to combine Anc80 with recently discovered transgenes and Selectas SVP-Rapamycin to create a novel gene therapy for MMA. This therapy is intended to a) enable the treatment of patients with and without pre-existing anti-AAV antibodies; b) prevent cellular immune responses that often reduce the expression levels of gene therapies; and c) provide the ability to administer repeat gene therapy doses to achieve sufficient levels of methylmalonyl-CoA mutase (MUT), the enzyme that MMA patients are lacking.

To advance the MMA program, Selecta entered into a Collaborative Research and Development Agreement (CRADA) with MEE and the National Human Genome Research Institute, NIH, in 2016. Principal investigators in this CRADA initiative are Charles Venditti, M.D., Ph.D., Senior Investigator and Head, Organic Acid Research Section in the National Human Genome Research Institute at the National Institutes of Health, and Luk Vandenberghe, Ph.D., Director of the Grousbeck Gene Therapy Center at MEE and an Assistant Professor at Harvard Medical School. A physician-scientist specializing in the study of inborn errors of metabolism including MMA, Dr. Venditti and his group have published several studies showing the effectiveness of gene therapy as a treatment for MMA in mice. Dr. Vandenberghe from MEE is the inventor of Anc80.

About Selecta Biosciences, Inc.

Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company that is focused on unlocking the full potential of biologic therapies by avoiding unwanted immune responses. Selecta plans to combine its tolerogenic Synthetic Vaccine Particles (SVP) to a range of biologics for rare and serious diseases that require new treatment options. The companys current proprietary pipeline includes SVP-enabled enzyme, oncology and gene therapies. SEL-212, the companys lead candidate in Phase 2, is being developed to treat severe gout patients and resolve their debilitating symptoms, including flares and gouty arthritis. Selectas clinical oncology candidate, LMB-100, is in a Phase 1 program targeting pancreatic cancer and mesothelioma. Its two proprietary gene therapy product candidates are being developed for rare inborn errors of metabolism and have the potential to enable repeat administration. The use of SVP is also being explored in the development of vaccines and treatments for allergies and autoimmune diseases. Selecta is based in Watertown, Massachusetts. For more information, please visit http://selectabio.com and follow @SelectaBio on Twitter.

Forward-Looking Statements

Any statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the company), including without limitation, whether the companys MMA product candidate will prevent cellular immune responses, enable repeat administration or allow for the treatment of patients with and without pre-existing anti-AAV antibodies, the companys ability to unlock the full potential of biologic therapies, the companys plan to apply its SVP platform to a range of biologics for rare and serious diseases, the potential of SEL-212 to treat severe gout patients and resolve their debilitating symptoms, the potential of the companys two gene therapy product candidates to enable repeat administration, the potential treatment applications for products utilizing the SVP platform in areas such as gene therapy, immuno-oncology, allergies, autoimmune diseases and vaccines, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors discussed in the Risk Factors section of the companys Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, or SEC, on May 11, 2017, and in other filings that the company makes with the SEC. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any obligation to update any forward-looking statements included in this press release.

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Selecta Biosciences Announces New Preclinical Gene Therapy Data at the Annual Meeting of the American Society of ... - GlobeNewswire (press release)

HIV has no cure. Its not quite the implacable scourge it was throughout the 1980s and 1990s, thanks to education, prophylactics, and drugs like PrEP. But still, no cure. Genetically-modified humans: what is CRISPR and how does it work?

Part of the problem is HIVs ability to squirrel itself away inside a cells DNA including the DNA of the immune cells that are supposed to be killing it. The same ability, though, could be HIVs undoing. All because of CRISPR. You know, CRIPSR: the gene-editing technique that got everyone really excited, then really sceptical, and now cautiously optimistic about curing a bunch of intractable diseases.

Last week, a group of biologists published research detailing how they hid an anti-HIV CRISPR system inside another type of virus capable of sneaking past a hosts immune system. Whats more, the virus replicated and snipped HIV from infected cells along the way. At this stage, it works in mice and rats, not people. But as a proof of concept, it means similar systems could be developed to fight a huge range of diseasesherpes, cystic fibrosis, and all sorts of cancers.

Those diseases are all treatable, to varying degrees. But the problem with treatments is you have to keep doing them in order for them to work. The current anti-retroviral therapy for HIV is very successful in suppressing replication of the virus, says Kamel Khalili, a neurovirologist at Temple University in Philadelphia and lead author of the recent research, published in Molecular Therapy. But that does not eliminate the copies of the virus that have been integrated into the gene, so any time the patient doesnt take their medication the virus can rebound. Plus treatments can and often do fail.

Gene therapy has promised to revolutionise medicine since the 1970s, when a pair of researchers introduced the concept of using viruses to replace bad DNA with good DNA. The first working model was tested on mice in the 1980s, and by the 1990s researchers were using gene therapies with limited success to treat immune and nutrition deficiencies. Then, in 1999, a patient in a University of Pennsylvania gene therapy trial named Jesse Gelsinger died from complications. The tragedy temporarily skid-stopped the whole field. Gene therapy had been steadily getting its groove back, but the 2012 discovery that CRISPR could make easy, and accurate, cuts on human genes, added more vigor.

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CRISPR as an agent for curing HIV has its own problems. For one, it has to be able to snip away the HIV from an infected cell without damaging any of the surrounding DNA. HIV mutates and evolves, so Khalili and his co-authors couldnt just program their CRISPR system with a single genetic mugshot. Instead, they had to target enough unchanging sections that were also critical to the virus survival.

Their next challenge was delivering the system to a critical mass of infected cells. First, you have to get it past the immune system which is programmed to attack any non-foreign object entering the body. They did this by packing their CRISPR system inside another type of virus called AAV (short for adeno associated virus). AAVs are a very small helper virus, they cant actually replicate in a cell on their own unless they have another virus there to help it along, says Keith Jerome, a microbiologist at the Fred Hutchinson Cancer Research Centre in Seattle. The great thing about AAVs is they cause essentially no immune system response in humans. Although thats not always true. Jesse Gelsinger died in 1999 because his immune system overreacted to the AAVs hed been given in his gene therapy trial. So doctors hoping to prescribe AAV-based gene therapy have to be aware of patients prior exposure.

In order to get approved for human use, this type of CRISPR-borne cure would have to be both safe and effective. This study got part of the way but was going strictly for efficacy: Does this work? Khalili and his co-authors treated mice and rat model with strains of HIV that were latent; hiding away in cellular DNAand others where the HIV was actively replicating. Then they used it on mice grafted with human cells. In all three cases, HIV rates went down significantly.

Other good news on the safety front: theres no evidence their trial made any off-target cuts. Ageing is a disease. Gene therapy could be the 'cure'

Theyll now need to run more experiments to make sure thats absolutely the case, probably using primate models since their DNA is closer to humans. They also have to make sure the treatment gets rid of enough HIV, so it doesnt just replicate itself back to harmful levels. In actual human patients theres no way that a CRISPR gene therapy will ever get 100 per cent of HIV, says Paul Knoepfler, a stem cell biologist at UC Davis. How highly efficient will be efficient enough to make a clinically meaningful impact?

Khalili believes he can get close enough. According to him, the CRISPR system doesnt need to eliminate all the HIV-infected cells, just enough so an HIV-patients immune system can get strong enough to take care of the rest on its own. I strongly believe in the gene-editing strategy, and with my 30 years in HIV research, I think this is the one that is going to take us to the end.

Hes not the only optimist. The advantage of using a virus as your delivery system is it can infect virtually every cell, says Jianhua Luo, a pathologist at the University of Pittsburgh. Luo is using a similar CRISPR-in-a-virus system to target cancerous DNA in cells.

And curing HIV could be a proof-of-concept for other diseases even genetic diseases people are born with. Although the virus starts as a simple infection, once it becomes part of a persons chromosome, it essentially becomes a genetic disease.

Since the HIV research was published, a team of biologists at University of California, Berkeley, described 10 new CRISPR enzymes that, once activated, are said to "behave like Pac-Man" to chew through RNA in a way that could be used as sensitive detectors of infectious viruses.

These new enzymes are variants of a CRISPR protein, Cas13a, which the UC Berkeley researchers reported last September in Nature, and could be used to detect specific sequences of RNA, such as from a virus. The team showed that once CRISPR-Cas13a binds to its target RNA, it begins to indiscriminately cut up all RNA making it "glow" to allow signal detection.

Two teams of researchers at the Broad Institute subsequently paired CRISPR-Cas13a with the process of RNA amplification to showed that the system, dubbed Sherlock, could detect viral RNA at extremely low concentrations, such as the presence of dengue and Zika viral RNA, for example. Such a system could be used to detect any type of RNA, including RNA distinctive of cancer cells.

Imagine a world where, instead of removing her breasts, Angelina Jolie could instead have taken a dose of genes that snip away the BRCA2 genes that threatened her with cancer. Thats the difference between a treatment and a cure.

Nick Stockton is a staff writer for WIRED US. This article originally appeared on WIRED. It has been updated to reference the new University of California, Berkeley research.

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CRISPR kills HIV and eats Zika 'like Pac-man'. Its next target? Cancer - Wired.co.uk