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Archive for the ‘Gene Therapy Research’ Category

Success of sensory cell regeneration raises hope for hearing restoration – Science Daily


Science Daily
Success of sensory cell regeneration raises hope for hearing restoration
Science Daily
In an apparent first, St. Jude Children's Research Hospital investigators have used genetic manipulation to regenerate auditory hair cells in adult mice. The research marks a possible advance in treatment of hearing loss in humans. The study appears in ...

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Success of sensory cell regeneration raises hope for hearing restoration - Science Daily

Prosper nonprofit holds fundraiser to help research cure for Hunter Syndrome – Nueces County Record Star

By Paulina De Alva, Prosper Press

The fifth annual Dancing With Dominic fundraiser, which benefits the research to find a cure for Hunter Syndrome, was hosted on Saturday, April 1, 2017 at Hughes Elementary School by the Henriquez family, whose 7-year-old son Dominic was diagnosed with the disease in October of 2011, when he was 22 months old.

The event included a dance in the gymnasium, performances by Prosper ISDs dance teams, face painting by the art students, a catered dinner, a silent auction, a raffle, kids activities and crafts, a photo booth and other activities for all the families.

Dominics mother, Jeanette Espinola, said she is incredibly thankful for the amount of community support shes seen during the planning process, and that the event was made possible all because of the help and support of the community of Prosper. She added that about 15 or 16 families from different parts of the country who have been affected by the disease attended the event.

We had a family gathering in conjunction with the event, she said. Because theres so few of us, were a very close community.

Hunter Syndrome, or Mucopolysaccharidosis Type II (MPS II), is a rare genetic disorder affecting 1 in 150,000 males that slowly destroys the bodys cells due to a missing enzyme, which results in the accumulation of cellular waste throughout the body. It is a progressive and life-limiting disease that mainly affects young boys, and the prognosis is that the children wont live past their teenage years. Espinola said it was a devastating diagnosis for her family.

All of a sudden you lose basically all your dreams that you had for your child, Espinola said. You were going to see him grow up, see him become an adult. But he may not make it past his teenage years.

She said the way she dealt with it was to do what she loves to do and plan to host a big fundraiser party along with her husband, who is a DJ, and with the help of the community in Virginia, where they lived before they moved to Prosper. Dominic loves to dance, so that party in 2012 became the first Dancing With Dominic event.

It was our way of contributing and helping find a cure, and bringing awareness, Espinola said. I think thats the other huge piece of it, is bringing awareness that there are these disorders, there are these kids, and that there is this potential right now to really help them, and the research is pretty much there, we just need the funds right now.

Dominics parents, Jeanette Espinola and Freddy Henriquez, founded the Hunter Syndrome Foundation in 2013, after having hosted already two Dancing With Dominic events, for the specific purpose of funding potential treatments and research and ultimately finding a cure for the disease.

There is one approved treatment for Hunter Syndrome. It consists of an infusion of the man-made version of the enzyme Dominic is missing, which is administered through a four-hour weekly IV treatment that prevents the disease from progressing fast. The medicine he gets, called Elaprase, costs about $12,000 per week, amounting to around half a million dollars per year, and is the second most expensive medicine in the country.

Hes been getting that for five years now since he was diagnosed, Espinola said. But the issue with that is that it doesnt cross into his brain. So he could still lose his cognitive skills, he could still begin regressing.

Its not a complete treatment, so for the past two years hes been in a clinical trial in Chicago where hes getting the enzyme to his brain. It helps in slowing down the progression of the disease in his brain.

Researchers have been searching for a permanent cure, so gene therapy is the next step they are working toward. The gene therapy research for Hunter Syndrome is led by two doctors, Douglas McCarty, Ph.D., and Haiyan Fu, Ph.D. at Nationwide Childrens Hospital in Columbus, Ohio. All of the Hunter Syndrome Foundation funds have benefited their work to find a cure. Dr. McCarty said the gene therapy for MPS II is the result of more than a decade of collaborative research efforts with support from MPS II patient family foundations.

This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier, McCarty said. Our preclinical data have shown great promise with lifelong benefits. We believe that we are well positioned to move forward towards a phase 1/2 clinical trial in patients with MPS II.

The vector for the gene therapy will cost $1.4 million to produce, and it will cost another million dollars to begin the clinical trials. The family donates the money raised from the yearly Dancing with Dominic event to the Hunter Syndrome Foundation, and through it, 100 percent of the funding goes toward the doctors research at Nationwide Childrens Hospital so they can find a cure.

Im not doing this by myself, there are families throughout the country who are also raising funds, Espinola said. So all of the families efforts put together throughout the country have raised over $500,000 so far to help the doctors in their research.

Espinola said she hopes the family-led efforts are able to fully fund the clinical trials for gene therapy and for some normalcy for her son, Dominic, in the future.

I hope that Dominic continues to do well and better treatments are found, she said. Maybe one day he can be an adult and lead somewhat of an independent life.

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Prosper nonprofit holds fundraiser to help research cure for Hunter Syndrome - Nueces County Record Star

Gene Therapy May Help Those With Hearing Loss – Healthline

Researchers may have brought us one step closer to gene therapy for the treatment of hearing loss, after discovering a way to regenerate auditory hair cells in mice.

It is estimated that about 15 percent of adults in the United States have some form of hearing loss, with men being twice as likely to develop the condition than women.

Damage to the auditory hair cells is one of the leading causes of hearing loss.

Aging is a common risk factor for such damage, although the ailment can also arise through prolonged exposure to loud noise, injury (such as head trauma), ear infections, and other illnesses and diseases.

Auditory hair cells are the tiny sensory cells of the cochlea the inner part of the ear that enable us to hear.

These cells consist of hair-like projections, called stereocilia, that are responsible for transforming sound vibrations into electrical signals that are sent to the brain.

In humans, auditory hair cells are unable to regenerate in order to replace damaged ones. In fish and birds, however, these cells can regenerate.

The process involves down-regulating expression of the protein p27 and up-regulating the expression of the protein ATOH1, notes study co-author Jian Zuo, Ph.D., of the Department of Developmental Neurobiology at St. Jude Childrens Research Hospital in San Francisco.

For their study published today in the journal Cell Reports Zuo and team set out to determine whether they could trigger the same process in mice.

Read More: Get the facts on age-related hearing loss

Using genetic manipulation, the researchers deleted the p27 protein and increased ATOH1 expression in mice.

When the mice experienced auditory hair cell damage as a result of exposure to loud noise, the researchers found that the cells supporting the auditory hair cells began to transform into auditory hair cells themselves.

Further investigation revealed that a number of proteins work together in order to regenerate auditory hair cells.

The researchers found that the deletion of p27 increased levels of a protein called GATA3 and boosted the expression of the POU4F3 protein. This increased ATOH1 expression, leading to auditory hair cell regeneration in the rodents.

The researchers explain that ATOH1 is a transcription factor required for the development of auditory hair cells. In humans, the production of ATOH1 stops in the womb.

According to Zuo and colleagues, however, their findings suggest that it may be possible to reactivate ATOH1 production in humans by genetically manipulating the p27, GATA3, and POU4F3 proteins.

Work in other organs has shown that reprogramming cells is rarely accomplished by manipulating a single factor," said Zuo. "This study suggests that supporting cells in the cochlea are no exception and may benefit from therapies that target the proteins identified in this study."

The researchers now plan to conduct a phase I clinical trial that will involve using gene therapy to reinstate ATOH1 production in humans.

The aim is to determine whether such a strategy can trigger auditory hair cell regeneration in humans, and whether this might be an effective treatment for hearing loss.

"Work continues to identify the other factors, including small molecules, necessary to not only promote the maturation and survival of the newly generated hair cells, but also increase their number," said Zuo.

Read More: What? Hearing loss expected to rise dramatically

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Gene Therapy May Help Those With Hearing Loss - Healthline

Scientists Find New Way to Fight HIV at Scripps Research Institute – NBC Southern California

Member-tethered, receptor-blocking antibodies protect cells from rhinovirus.

A new approach to treating AIDS was discovered by scientists at the Scripps Research Institute (TSRI).

Scientists have found a way to stick HIV-fighting antibodies to immune cells, which may foster a cell population resistant to the virus.

The experiments under lab conditions show resistant cells can quickly replace diseased cells under lab conditions, which shows the potential to cure a person with HIV, according to TSRI.

"This protection would be long term," said Jia Xie, senior staff scientist at TSRI and the first author of the study. It was published Monday in the journal Proceedings of the National Academy of Sciences.

Richard Lerner, M.D., Lita Annenberg Hazen Professor of Immunochemistry at TSRI, led the study. The researchers will work with investigators at City of Hope's Center for Gene Therapy to investigate this new therapy as a potential treatment for HIV.

They will evaluate the treatment with safety tests as required by federal regulations.

"City of Hope currently has active clinical trials of gene therapy for AIDS using blood stem cell transplantation, and this experience will be applied to the task of bringing this discovery to the clinic," said John A. Zaia, M.D., director of the Center for Gene Therapy, in a statement.

"The ultimate goal will be the control of HIV in patients with AIDS without the need for other medications," said Zaia.

A significant new advantage with this treatment is that antibodies hang onto a cell's surface, blocking HIV from accessing a crucial cell receptor and spreading infection, according toTSRI.

This is really a form of cellular vaccination, said Lerner.

Antibodies recognize the CDR4 binding site, which allows them to block HIV from attacking a critical receptor in the cell. Scientists say this technique can produce an HIV-resistant population of cells.

Published at 7:14 PM PDT on Apr 10, 2017 | Updated at 7:15 PM PDT on Apr 10, 2017

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Scientists Find New Way to Fight HIV at Scripps Research Institute - NBC Southern California

Brain cells reprogrammed to make dopamine, with goal of Parkinson’s therapy – The San Diego Union-Tribune

In a pioneering study, European scientists have reprogrammed brain cells in mice to correct some of the movement disorders of Parkinsons disease.

The scientists also demonstrated the reprogramming in human brain cells grown in cultures.

In both mice and human cell cultures, the procedure converted brain cells called astrocytes into cells that produce dopamine, a neurotransmitter necessary for movement. Dopamine-making neurons are destroyed in Parkinsons disease; so replacing them should alleviate symptoms.

Like all biomedical research, this approach will require more development and testing before it can be considered for treating actual patients.

The study was published Monday in Nature Biotechnology. Pia Rivetti di Val Cervo was first author, and Ernest Arenas was senior author. Both are of Karolinska Institute in Stockholm, Sweden.

The study can be found online at j.mp/astropark.

Researchers worked on mice that had had their dopamine-making neurons destroyed. They used a viral delivery system to transmit three genes to the astrocytes that reprogrammed some of them into dopamine-making cells.

The next steps to be taken toward achieving this goal include improving reprogramming efficiency, demonstrating the approach on human adult striatal astrocytes, developing systems to selectively target human striatal astrocytes in vivo, and ensuring safety and efficacy in humans, the study concluded.

The study is a more sophisticated version of gene therapy approaches that have previously been investigated for Parkinsons, and is worth pursuing, said Parkinsons disease researcher Andres Bratt-Leal. However, much more work needs to be done before it can be considered for patients, he said. Meanwhile, other therapeutic projects are much closer to clinical testing.

Bratt-Leal is involved in one of those projects, a San Diego-based initiative to reprogram skin cells from Parkinsons patients into embryonic-like cells called induced pluripotent stem cells, and then mature them into the dopamine producing neurons. These neurons will then be implanted into the brains of the patients, if work by the Summit for Stem Cell Foundation succeeds.

Implanting new neurons has shown tremendous promise in animal models and clinical trials using dopamine-producing neurons derived from embryonic stem cells or induced pluripotent stem cells are going to start in the next 1 to 2 years, said Bratt-Leal, the foundations director of research. Gene therapy is promising, but there remain a lot of questions before it is ready for clinical trial.

In a dish, only a fraction of the cells are successfully made into cells which resemble dopamine-producing neurons, Bratt-Leal said. I'd like to know what happens to all the other cells which don't complete that transformation. Are the cells made with gene therapy as good as the neurons we can make from stem cells?

With cell therapy clinical trials around the corner and improvements in gene therapy technology, patients with Parkinson's disease have reasons to stay active and optimistic about the future.

bradley.fikes@sduniontribune.com

(619) 293-1020

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Brain cells reprogrammed to make dopamine, with goal of Parkinson's therapy - The San Diego Union-Tribune

Research Reveals Targeted Gene Therapy Is More Advantageous … – Digital Journal

A research report by published in the Journal of medical informatics on the topic of Research Progress on Treatment of Cancer with Compatibility of Traditional Chinese Medicine establishes that the targeted gene therapy is more effective than Surgery, Chemotherapy and Radiotherapy.

Cancer is one of the major life-threatening diseases that people often worry about. People suffering from cancer often undergo traditional treatments, such as surgery, chemotherapy and radiotherapy. However, such treatments could have harmful side effects. A research was recently conducted aimed at studying traditional Chinese medicine compatibility with respect to treating prostatic cancer. The study reveals that the targeted gene therapy is more advantageous to traditional cancer treatments of Surgery, Chemotherapy and Radiotherapy.

The study also points out the targeted gene therapy can be combined with other therapies for a more effective result. However, this therapy is also not mature enough to address all health issues related to the prostatic cancer. In such a scenario, Dr. Songs 3D Prostate Targeted Treatment emerges as a safe and reliable treatment for prostate cancer. The research revealed that the therapy can be combined with local targeted injections. The technique makes the therapy more effective and increases its killing effect on cancer cells.

Video Link: http://www.youtube.com/embed/xIFCz5p8PDo

Dr. Songs treatment is based on the direct injection technique of the traditional Chinese medicine system. Dr. Xinping Song acknowledges the findings of the research and also the anti-cancer extract compatibility of the traditional Chinese medicine.In this prostate cancer treatment, patients are given small targeted injections in the affected areas of the prostate to help eliminate causative pathogens and clear the blockage. The injections carry herbal extracts only, which are safe and have no side effects at all. This is the reason this treatment is harmless and more effective than surgery or chemotherapy.

Dr. Song believes that the traditional Chinese anticancer medicine can better interpose with the symptoms of cancer patients. Dr. Songs prostate cancer treatment that follows the principles of traditional Chinese medicine is a clinical breakthrough. This innovative treatment brings more advantages in patients and their familys lives.

At Dr. Song 3D Urology and Prostate Clinic, patients can undergo all types of prostate care and treatment, including the treatment for the prostate cancer. This natural treatment method saves the cost and also meets the patients requirements in an effective manner. With a non-surgical and quality treatment, patients gradually improve their health and get rid of their pain and sufferings.

To know more about Dr Songs 3D prostate treatment, one can visit the website https://www.prostatecancer.vip/

About 3D Urology and Prostate Clinic

The 3D Urology and Prostate Clinic is a premier prostate treatment clinic. The clinic specializes in treating various types of prostate diseases and complications, such as prostatitis, enlarged prostate, benign prostatic hyperlasia (BPH), prostate cancer, seminal vesiculitis, epididymitis,cystitis, prostate blockage and calcification, and chronic pelvic pain syndrome (CPPS), etc. The clinic is a medical clinic, licensed and approved by the Ministry of Health of the Peoples Republic of China.

Media Contact Company Name: Dr. Song's 3D Prostate and 3D Prostatitis Clinic Contact Person: Miss Alisa Wang, English Assistant to Dr. Song Email: prostatecure3d@gmail.com Phone: 86-186-7321-6429 Address:The Xiangtan 3D Prostatitis and Prostate Clinic Address - Jin Xiangtan Square Office Building, Suite 801, Shao Shan Middle Rd City: Xiangtan State: Hunan Country: China Website: https://www.prostatecancer.vip/

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Research Reveals Targeted Gene Therapy Is More Advantageous ... - Digital Journal

Gene research for Essex teenager with rare wasting disease could be a world first – Dunmow Broadcast

PUBLISHED: 08:45 06 April 2017

Angela Singer

Maddi Thurgood pictured on the farm at Saffron Walden County High

Archant

Scientists are now being recruited for research posts to work on a gene therapy strategy for Maddi Thurgood, the teenager whose rare, so far incurable, wasting condition they hope to treat. If it works, it will be a world first.

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Having travelled to America and Canada to see top specialists, help is now being offered by Sheffield University of Neurosciences (SITRAN) but it will cost 224,000.

With the community rallying in both Saffron Walden and Dunmow, over half that sum has been raised. Maddie is a pupil at the Joyce Frankland Academy in Newport and was previously at Helena Romanes in Dunmow.

After this latest medical trial was reported in The Saffron Walden Reporter and The Dunmow Broadcast, with a picture of Maddie receiving 700 presented by The Ohio Country Music Club in Newport, another benefactor has given 10,000.

Melissa Jones, captain of Saffron Walden Golf Club, is a trustee of The Donald Forrester Trust, set up by her late uncle.

She said: I was aware of the campaign and the collection jars in the shops round the town and when I saw the picture of the donation, I thought our trust was set up for this purpose.

Maddi was diagnosed in April 2016, just after her 15th birthday, with spastic paraplegia gene 15. Its a motor neurone so rare, no one else is known to have it in the UK and fewer than 20 people worldwide. However the Sheffield research project, to develop a gene therapy just for Maddi, could also help a three-year-old girl called Robbie in Boston, America.

Maddis mum, Carina spends her days researching across the globe in a race against time for something to stop her daughter deteriorating.

The youngster, once a keen ice-skater and still an enthusiastic pupil on the animal welfare course at Saffron Walden County High, now walks with a stick. She goes to school on her good days and is always seen with a smile.

Carina, said: We are in touch with Robbies parents constantly. We didnt choose to be in this situation but we are trying to do the best for our children.

The condition eventually affects all four limbs, the brain, vision and hearing. A website: saveourmaddi.co.uk has been set up to appeal for fundraisers. To help Maddi, email: info@saveourmaddi.co.uk or see: http://www.facebook.com/SaveOurMaddiAppeal or http://www.treeofhope.org.uk/maddis-story-save-our-maddi

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Gene research for Essex teenager with rare wasting disease could be a world first - Dunmow Broadcast

World-renowned scientists to speak at Precision Medicine in Child Health research day – UCalgary News

Who would have ever thought that some forms of blindness could be repaired? Ian MacDonald is a pioneer in gene therapy as a treatment of genetic eye diseases. The ophthalmologist will be one of two external speakers at the Alberta Childrens Hospital Research Institute (ACHRI) symposium Wednesday, April 19 at the Foothills Campus. MacDonald, a professor in the Department of Opthalmology and Visual Sciences at the University of Alberta, will speak on Novel Therapeutic Approaches to Human Disease The Example of Ocular Gene Therapy. We spoke to him briefly about his research.

Q: Why are ocular diseases at the forefront of precision medicine?

A: The eye is an attractive target for precision medicine and gene therapy as it offers a readily accessiblesite for surgical intervention and injection, is relatively immune-privileged, andtreatment of only one eye allows the non-treated eye to serve as a control for the experimental therapy.

Q:Is this the right time to pursue research in precision medicine?

A: Now is definitely the right time to pursue research in precision medicine. Phenotyping in human ocular heritable disease is advanced and we can now make clinical decisions backed up by molecular genetic confirmation. With new tools ofnext generation sequencing, we have a lot to offer in terms of precision medicine to our patients and families.

Q:How important is basic research to your accomplishments?

A:The first ocular gene therapy trial for choroideremia was based on the products of 30 years of scientific research (somein Canada, including mapping the gene in 1987). It simply could not have occurred without solid pre-clinical research, a team of informed and talented researchers, and significant infrastructure and research support from national (CFI, CIHR, FFB Canada, Choroideremia Research Foundation, Canada Inc.) and provincial (AIHS) funding agencies.

Fruit flies model how human traits are passed on

Trudy Mackay is a distinguished scientist specializing in quantitative genetics. Her research relies on the fruit fly an insect that has provided scientists the means for biomedical research and discovery for over 100 years. Mackays work has allowed researchers across the world to understand the genetic traits crucial to plant, animal and human health. A fellow of the Royal Society and the National Academy of Sciences, Mackay was awarded the 2016 Wolf Prize Laureate for Agriculture, widely considered one of the most prestigious prizes in science. She is now at North Carolina State University. Mackay will speak on The Genetic Architecture of Complex Traits: Lessons From Drosophila. We also spoke to her briefly about her research.

Q:Is fruit fly DNA much different from ours?

A:The fruit fly genome is about 10 times smaller than the human genome. However, and perhaps surprisingly, about two-thirds of fly genes have a human counterpart, and 70 per cent of human disease genes have a fruit fly counterpart. Thus, flies are a good genetic model for quantitative traits, including diseases, in humans.

Q:The first human genome was sequenced in 2003. How much more complex are human genes than originally thought?

A:The first human genome sequence was surprising in that many fewer genes were present than experts had predicted. We now know that regulatory component of the human genome is at least as important as the protein coding genes, and deciphering the regulatory code is an active area of research.

Q:How important is precision medicine to our future and health care of children?

A:Precision medicine is "an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person." Precision medicine has the promise to revolutionize future heath care by using genetic and genomic data to optimize individual disease risk assessment and therapy.

UCalgary and ACHRI researchers to present at symposium

The symposium will also hear from University of Calgary and ACHRI researchers:

Information on registration to attend the Alberta Childrens Hospital Research Institute Symposium on Precision Medicine in Child Health and details on the program agenda are available on the ACHRI website.

The Alberta Childrens Hospital Research Institute (ACHRI) symposium is an annual event supported by generous community donations through the Alberta Childrens Hospital Foundation. The research day highlights leading child health research from pre-conception to early adulthood. The day consists of presentations from external and internal speakers and a poster competition, TED Talks and presentations from ACHRI trainees. The symposium concludes with presentation and poster awards from ACHRI Director Dr. Brent Scott.

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World-renowned scientists to speak at Precision Medicine in Child Health research day - UCalgary News

Australasian Gene Cell Therapy Society AU

Dear AGCTS Membership & Associates,

At our 2014 Annual General Meeting, we unanimously voted for a name change to the Australasian Gene and Cell Therapy Society (AGCTS) to reflect the broader interests of our society members in cell biology, particularly the use of stem cells in delivering gene medicines. The name change of our Society was finalised in 2015, announced to our membership and associates via email. You are now looking at our new website http://www.agcts.org.au.

At that meeting, the Executive Committee heard the membership loud and clear. Stronger links must be forged with cell therapy and stem cell research community. To achieve this goal we have partnered with the Australasian Society for Stem Cell Research (ASSCR) to deliver what is shaping up to be an inspiring and exciting Scientific Program with an incredible line-up of international research leaders and clinicians 24th 26th May 2017, UTS Aeriel Function Centre, Sydney.

I would like to acknowledge the already enormous contribution of the Joint 10th AGCTS and ASSCR Conference Organising Committee who have volunteered their time to make this meeting a success. Id like to individually thank our AGCTS VP Jim Vadolas, Treasurer Ann Simpson, Secretary Samantha Ginn, Exec Members Paul Gregorevic, Sharon Cunningham, Marguerite Evans-Galea, Ex-officio member Steve Wilton and ASSCR President Melissa Little, past-President Michael OConnor, VP Ed Stanley, Treasurer Robyn Meech, Secretary Michael Morris, Exec Members Michael Doran, Margret Schuller, Helen Abud, Trish Barker.

I am very proud to be leading a dedicated society which has continued to provide advocacy for Australian research in the development of molecular medicines, cell and gene therapies. Our membership works tirelessly to reduce the impact of some of Australias largest health problems and improve the well being of those diagnosed with acquired or genetic disease. Focus areas of our membership are broad and include cancer, viral infection (HIV, Hepatitis B & C), autoimmunity, immunodeficiency, diabetes, metabolic disease, blood, eye, ear and muscle disorders.

Although there have been considerable funding challenges of late, momentum is clearly building thanks to the enormous commitment and perseverance and of our researchers, clinicians, industry leaders and regulators. The AGCTS Executive Committee and I feel privileged to represent you and our sector in this very exciting journey and witness first-hand the translation of our field into the clinic which will be featured at our next meeting.

I look forward to welcoming you to our Joint 10th AGCTS and ASSCR Scientific Meeting in Sydney, 24th 26th May 2017.

Kind regards,

Rosetta Martiniello-Wilks PhD

President, Australasian Gene and Cell Therapy Society http://www.agcts.org.au

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Australasian Gene Cell Therapy Society AU

Sarepta nabs Regeneron exec Stehman-Breen as new CMO – FierceBiotech

As it continues its sales push for the controversial Duchenne med Exondys 51 (eteplirsen), Sarepta has taken on a new chief medical officer to help it develop its next-gen DMD pipeline.

The biotech announcedthis morning that Catherine Stehman-Breen, M.D., M.S., has become its new CMO after her two-year stint as VP of clinical development and regulatory affairs at Regeneron, and coming after a 12-year tenure at Amgen, where she led the neuroscience, nephrology and bone therapeutic areas.

She will take the role away from Ed Kaye, M.D., the companys CEO, who had also been holding the dual position of chief medical officer since 2011.

I deeply admire Sareptas profound commitment to improving the lives of boys with Duchenne muscular dystrophy and the exciting and innovative PMO and PPMO platform that is being harnessed to achieve this goal, said Stehman-Breen.I am excited to join the company at a time when it is rapidly building and look forward to working closely with the internal team and external collaborators as we seek to develop and commercialize novel therapies that address this significant unmet medical need.

We are thrilled to have Dr. Stehman-Breen join Sarepta and our mission to develop treatments for boys with Duchenne muscular dystrophy, added Kaye. Her extensive experience in global development, clinical operations and research across multiple therapeutic areas, at leading biopharmaceutical companies, positions her well to lead our medical teams and rapidly advance our RNA-targeted platforms and gene therapy programs.

Its current FDA-approved DMD med, which got the nod last fall despite having limited data and a negative AdComm, can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD.

Its pipeline is now trying to treat more boys with the genetic condition that will usually prove fatal in early adulthood, and includes research deals with Nationwide Childrens Hospital to work on their microdystrophin gene therapy program, as well as another form of gene therapy.

An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Childrens.

It has also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin.

The goal here will be to produce a muscle cell that can function normally even when dystrophin is absent.

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Sarepta nabs Regeneron exec Stehman-Breen as new CMO - FierceBiotech

New Gene Therapy for Cancer Offers Hope to Those With No Options Left – NBCNews.com

Dimas Padilla, 43, of Kissimmee, is in remission from non-Hodgkin's lymphoma after receiving an experimental cancer therapy called CAR-T. Here, he poses with his wife, Dimas Padilla. NBC News

"These are patients who really are without hope," Locke said.

"Patients who at best could expect to have a one in 10 chance of having a complete disappearance of their lymphoma," he added. "So the results are really exciting and remarkable."

More than 80 percent of the 101 patients who got the treatment were still alive six months later. "Only about half the patients who (went) on this study could expect to even be alive six months after the therapy," Locke said.

Padilla is one of them. When the cancer came back most recently time, his lymph nodes were bulging. "They were so bad that they moved my vocal cords to the side and I was without my voice for almost three months," he said.

"They kept growing and my face was swelling, and I thought I was going to choke while I was sleeping."

Padilla was among the last patients enrolled in the trial.

"Once they infused the cells in my body, within two to three days all my lymph nodes started melting like ice cubes," he said.

The treatment is no cake walk. Just as with a bone marrow transplant, the patient's immune system must be damaged so that the newly engineered T-cells can do their work. That involves some harsh chemotherapy.

It's so harsh that it killed three of the patients in the trial. Padilla says he still has some memory loss from his bout with the chemo.

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"I had some fevers and I was shaking and a little bit of memory loss but it was temporary," he said. "I will say that it was pretty intense for like a week, but in my second week, second week and a half, I was starting to feel more normal. I was able to start walking and the shaking was not as bad as it was in the beginning," he said.

And when he got the news that his lymphoma was gone at least for now Padilla was delighted.

"I kissed my wife. I probably kissed the doctor," he said.

The company developing the treatment, Kite Pharma, sought Food and Drug Administration approval for the therapy on Friday.

It carries the tongue-twisting name of axicabtagene ciloleucel, and it's the first commercial CAR-T product to get into the FDA approval process.

It's far too early to say any of the patients were cured, Locke cautions. And such a difficult treatment course is really only for patients in the most desperate condition.

"The patients in this trial were really without options," he said.

But Locke is sold on the approach. "This is a revolution. It's a revolution in cancer care. This is the tip of the iceberg," he said.

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New Gene Therapy for Cancer Offers Hope to Those With No Options Left - NBCNews.com

Prosper nonprofit brings gene therapy treatment closer to reality – Star Local Media

Over the past three years, parent-led efforts have collectively raised half a million dollars to support gene therapy research at Nationwide Childrens Hospital (Columbus, Ohio) to treat the ultra-rare disease Hunter Syndrome (also known as Mucopolysaccharidosis or MPS II).

The research is led by Dr. Douglas McCarty and Dr. Haiyan Fu of Nationwide, and funding is expected to bring the research closer to a human clinical trial in late 2017 or early 2018.

To raise funds, the parent-led foundations organize local fundraisers. In Prosper, the Henriquez family hosts an annual family event called Dancing with Dominic, in honor of their 7-year old son Dominic who suffers from Hunter Syndrome. Dominic is a 1st grader at Baker Elementary in Prosper ISD. Without a cure or emerging treatment, Dominic may not live past his teenage years.Dancing with Dominic 2017 will be held on April 1, at Hughes Elementary in McKinney, Texas. Dr. McCarty and Dr. Fu are among the hundreds that plan to attend the event this year.

Hunter Syndrome is a rare, genetic condition that affects approximately 2,000 patients worldwide, almost exclusively young boys. Patients are missing an enzyme, resulting in the accumulation of cellular waste throughout the body.

Babies develop normally for the first few years, and then begin to experience progressive symptoms like stiff joints, enlarged liver and spleen, behavioral problems, constant ear infections and runny nose, and heart valve complications. The average life span for someone with the most common, severe form of the disease is in the early teens.

There is no cure for the disease, although once diagnosed, patients can begin receiving a weekly 4-hour infusion of an enzyme replacement therapy. This medication, Elaprase, is one of the most expensive in the world, often $500,000 or more per patient, per year, and only stabilizes some of the physical symptoms of the disease. Because it does not cross the blood-brain-barrier, it does nothing to prevent the progressive brain damage that occurs in most children affected by the disease.

This gene therapy for MPS II is the result of more than a decade of collaborative research efforts with support from MPS II patient family foundations, Dr. McCarty said. This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier. Our preclinical data have shown great promise with lifelong benefits. We believe that we are well positioned to move forward towards a Phase 1/2 clinical trial in patients with MPS II.

About the Hunter Syndrome

Foundation

The Hunter Syndrome Foundation is a 501(c)3 non-profit corporation with a mission to fund potential therapies that will ultimately find a cure for this disorder.

The Foundation is part of a coalition of parent-led organizations that are supporting medical research for hunter syndrome. The Foundation was established in Prosper, Texas by the Henriquez family. For more information, visit http://www.huntersyndromefoundation.org.

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Prosper nonprofit brings gene therapy treatment closer to reality - Star Local Media

Cutting-edge gene therapy provides hope for patients with inherited eye conditions – Miami Herald


Miami Herald
Cutting-edge gene therapy provides hope for patients with inherited eye conditions
Miami Herald
The trial will evaluate a cutting-edge concept: gene therapy. While there are no gene therapy products currently approved for use in the U.S., researchers are experimenting to see if they can provide a solution to alleviate hereditary diseases. Gene ...

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Cutting-edge gene therapy provides hope for patients with inherited eye conditions - Miami Herald

Faster way to test new gene therapies – Cordis News

The development of gene therapy vectors for the hereditary immunodeficiency Chronic Granulomatous Disease (CGD) is hampered by the absence of human cell lines, necessary for rapid and effective gene therapy vector testing. A new model supported by EU funding can make the process more efficient.

CI Photos, Shutterstock

When no donor matches, a few locations globally will carry out gene therapy. But for this to be used clinically, the efficacy of the treatment has to be established. The testing process depends on cellular models and currently the process is labour intensive, lengthy and expensive.

A team of researchers headed by Dr Janine Reichenbach, a professor and Co-Head of the Division of Immunology at the University Children's Hospital Zurich, has developed a new cellular model that enables researchers to test the efficacy of new gene therapies much more efficiently.

We used Crispr/Cas9 technology to change a human cell line so that the blood cells show the genetic change typical of a specific form of Chronic Granulomatous Disease, explains Professor Reichenbach.

The standard way of testing has been to use patients skin cells that are reprogrammed into stem cells, a time consuming and expensive process. The new testing system will be more cost effective. Our system makes the process faster and cheaper which means well be able to develop new gene therapies for affected patient more efficiently, she adds.

In more detail, to test the vectors of potential benefit to people suffering from p47phox-deficient chronic granulomatous disease (CGD), the article published in the journal Scientific Reports explains researchers have generated a cellular model consisting of regularly interspaced, short palindromic repeats (CRISPR)/Cas9. This introduces a GT-dinucleotide deletion (GT) mutation in p47phox encoding NCF1 gene in the human acute myeloid leukemia PLB-985 cell line.

The research has also lead to a different approach to the transfer of healthy copies of the gene into the affected cells. So far therapies have used modified, artificial viruses as transporters, but some patients went on to develop cancer so this first generation of viral correction systems is now outdated. Dr Reichenbachs team now use lentiviral, self-inactivating gene therapy that she describes as more efficient and safer.

She believes these to be an intermediate stage only, and looks forward to the future use of genome editing to provide greater precision. However, Science Daily suggests this will take another five to six years before precision gene surgery is available clinically.

An exciting approach to treatment furthered by EU support

CELL-PID (Advanced cell-based therapies for the treatment of Primary ImmunoDeficiency) and NET4CGD (Gene therapy for X-linked Chronic Granulomatous Disease - CGD) are two projects that have received funding from the EU. The University Children's Hospital Zurich is one of three European centers able to use this new gene therapy in an international clinical phase I/II study to treat patients with Chronic Granulomatous Disease as part of NET4CGD.

For more information, please see: NET4CGD project website CELL-PID CORDIS project webpage

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Faster way to test new gene therapies - Cordis News

Horae Gene Therapy Center UMass Medical School

Treating human diseases by utilizing gene therapy strategies have taken the scientific world by storm. Imporved delivery tools (i.e. AAVs) and novel therapeutic strategies are prooving that gene therapy has the promise of successfully threating a wide spectrum of diseases that were once uncurable. Read more about what is gene therapy, what are the tools and current strategies scientists use to advance the field and cure disease.

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The Faculty of the Horae Gene Therapy Center is dedicated to develop therapeutic approaches for rare inherited diseases for which there is no cure utilizing state of the art technologies to correct the genetic mutations.Our focus is on AAT Deficiency,Amyotrophic Lateral Sclerosis (Lou Genrig's Disease),Canavan Disease,Cystic Fibrosis,Tay-Sachs&Sandhoff diseases,Retinitis Pigmentosa,Huntington's disease,HypercholesterimiaandCardiac arrhythmia.

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Under the umbrella of the ATC, UMass Medical School formed the Gene Therapy Center (GTC) emphasizing the promise that lies within the application of the recombinant adeno-associated virus;RNA Therapeutics Institute (RTI)featuring novel strategies for using the RNAi mechanism to silence the action of individual genes and The Center for Stem Cell Biology and Regenerative Medicine seeking to unlock the enormous promise to elucidate disease mechanisms inherent in humans.

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The Horae Gene Therapy Center is always interested in possible partnerships with both indivituals and organizations.Some possible partnerships includeknowledge exchange,consulting,collaborations with academia and/or industry,reagent exchange,sponsored research, andspinout activities.We encourage you to contact us and learn more how we can partner together to advance the area of gene therapy.

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Horae Gene Therapy Center UMass Medical School

Pioneering stem cell gene therapy cures infants with bubble baby disease – UCLA Newsroom

FINDINGS

UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.

In a phase 2 clinical trial led by Dr. Donald Kohn of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchat UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohns treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the childs own cells, has cured 30 out of 30 babies during the course of several clinical trials.

Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.

Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzyme a lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipients body rejecting the transplanted cells.

The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patients own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each childs bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.

The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.

The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.

The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.

Kohn is a professor of pediatrics and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA and member of the UCLAChildrens Discovery and Innovation Institute at Mattel Childrens Hospital. The first author of the study is Kit Shaw, director of gene therapy clinical trials at UCLA.

The research was published in the Journal of Clinical Investigation.

The research was funded by grants from the U.S. Food and Drug Administrations Orphan Products Clinical Trials Grants Program (RO1 FD003005), the National Heart, Lung and Blood Institute(PO1 HL73104 and Z01 HG000122), the California Institute for Regenerative Medicine (CL1-00505-1.2 and FA1-00613-1), the UCLA Clinical and Translational Science Institute (UL1RR033176 and UL1TR000124) and the UCLA Broad Stem Cell Research Center.

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Pioneering stem cell gene therapy cures infants with bubble baby disease - UCLA Newsroom

Stamford-Based Cell And Gene Therapy Nonprofit Plans New York Gala – Stamford Daily Voice

STAMFORD, Conn. Stamford-based Alliance for Cancer Gene Therapy (ACGT), the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer, will celebrate its anniversary with a gGala on Wednesday, April 19, 2017, at 6:30 p.m. at The Harvard Club of New York City.

Dr. John Lahey, president of Quinnipiac University, will be honored at the event with the first ever Edward Netter Award for Business and Industry for his contributions to ACGT and the community. Gala speakers also include: ACGT research fellow Dr. Robert Vonderheide of the University of Pennsylvania, who will speak on the exciting breakthroughs using immunotherapy for the treatment of solid cancers; and Doug Olson, one of the first three patients treated in the groundbreaking cancer immunotherapy CAR-T clinical trial developed by ACGT research fellow and Scientific Advisory Council member, Dr. Carl June.

Tickets for the ACGT Gala are $750 and are available at acgtfoundation.org/events/ or by emailing Barbara Gallagher, ACGT national director of philanthropy at bgallagher@acgtfoundation.org.

Barbara Netter of Greenwich, ACGTs honorary chairman of the Board and co-founder, will present the first-ever Edward Netter Award for Business and Industry to Dr. Lahey, an ACGT Board member since 2004. Dr. Lahey embodies the qualities prized by ACGT co-founder Edward Netter: intellect, creativity, tenacity, curiosity and compassion.

He is the eighth President of Quinnipiac University, a private university located in Hamden. Upon his arrival at Quinnipiac in March of 1987, Dr. Lahey initiated a strategic planning process that has resulted in the growth of student enrollment from 2,000 to nearly 10,000 students. He also expanded Quinnipiac from a college to a university, which now offers more than 100 programs in its nine schools and colleges: Arts and Sciences, Business, Communications, Education, Engineering, Health Sciences, Law, Medicine and Nursing.

ACGT research fellow, Dr. Robert Vonderheide, will speak about his current work in immunotherapy that is targeting pancreatic cancer and other solid tumors at the Abramson Cancer Institute at the University of Pennsylvania. Dr. Vonderheides translational work tests novel approaches such as vaccines, antibodies, and adoptive T cells for the treatment of patients with melanoma, pancreatic cancer and other cancers. ACGT funded Dr. Vonderheides early research work in immunotherapy.

Doug Olson of Tinicum Township, Pa. will share the details of his experience as patient #2 in the first CAR-T 19 immunotherapy trial in September 2010 at the University of Pennsylvania. The treatment for the trial was developed by ACGT research fellow, Dr. Carl June. Doug was diagnosed with chronic lymphocytic leukemia (CLL) in July 1996 and has remained in remission since completing the trial.

This Gala offers the opportunity to celebrate the successes of ACGT research fellows and honor those who make it all possible, said John Walter, CEO and president of ACGT. With our donors support, ACGT has been able to be a part of finding and funding some of the most exciting cancer treatment breakthroughs, several of which we anticipate coming to market this year.

ACGt was founded in 2002 by Greenwich residents Barbara and Edward (1933-2011) Netter. As a national non-profit, ACGT has provided nearly $27 million in funding for cancer cell, gene and immunotherapy research in North America.

Photo: The Alliance for Cancer Gene Therapy (ACGT), gala event committee is (left to right) Sharon Phillips, Margaret Cianci, ACGT executive director, Barbara Gallagher, Jacquie Walter, Barbara Netter, ACGT co-founder and event chair, John Walter, ACGT CEO and president, Martha Zoubek, Jenifer Howard and Tracy Holton. Not pictured is Sabrina Raquet.

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Stamford-Based Cell And Gene Therapy Nonprofit Plans New York Gala - Stamford Daily Voice

Promising anti-aging gene therapy developed through innovative … – UH System Current News

From left: Richard Allsopp, David Watumull and Bradley Willcox

Promising anti-aging results have been shown by a study of the compound Astaxanthin by researchers at the University of Hawaii John A. Burns School of Medicine in partnership with the life sciences company Cardax Inc.

The Astaxanthin compound CDX-085, developed by Cardax, showed the ability to significantly increase the expression of the FOXO3 gene, which plays a proven role in longevity.

All of us have the FOXO3 gene, which protects against aging in humans, said Bradley Willcox, professor and director of research at the Department of Geriatric Medicine, JABSOM, and principal investigator of the National Institutes of Health-funded Kuakini Hawaii Lifespan and Healthspan Studies. But about one in three persons carry a version of the FOXO3 gene that is associated with longevity. By activating the FOXO3 gene common in all humans, we can make it act like the longevity version. Through this research, we have shown that Astaxanthin activates the FOXO3 gene, said Willcox.

Richard Allsopp in his lab

This preliminary study was the first of its kind to test the potential of Astaxanthin to activate the FOXO3 gene in mammals, said Richard Allsopp, associate professor, and researcher with the JABSOM Institute of Biogenesis Research.

In the study, mice were fed either normal food or food containing a low or high dose of the Astaxanthin compound CDX-085 provided by Cardax. The animals that were fed the higher amount of the Astaxanthin compound experienced a significant increase in the activation of the FOXO3 gene in their heart tissue.

We found a nearly 90 percent increase in the activation of the FOXO3 Longevity Gene in the mice fed the higher dose of the Astaxanthin compound CDX-085, said Allsopp.

This groundbreaking University of Hawaii research further supports the critical role of Astaxanthin in health and why the healthcare community is embracing its use, said David G. Watumull, Cardax CEO. We look forward to further confirmation in human clinical trials of Astaxanthins role in aging.

We are extremely proud of our collaborative efforts with Cardax on this very promising research that may help mitigate the effects of aging in humans, said UH Vice President of Research Vassilis L. Syrmos. This is a great example of what the Hawaii Innovation Initiative is all aboutwhen the private sector and government join forces to build a thriving innovation, research, education and job training enterprise to help diversify the states economy.

Read more about the research at the JABSOM website.

See more images from the JABSOM-Cardax press conference.

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Promising anti-aging gene therapy developed through innovative ... - UH System Current News

Could gene therapy, with help from California’s stem cell agency, treat ‘bubble boy disease’? – San Francisco Business Times


San Francisco Business Times
Could gene therapy, with help from California's stem cell agency, treat 'bubble boy disease'?
San Francisco Business Times
Dr. Morton Cowan is the principal investigator of a gene therapy trial at UCSF that is more. University of California Television. CIRM funding gives UCSF, St. Jude researchers a chance to show that combining gene therapy with a chemotherapy agent ...

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Could gene therapy, with help from California's stem cell agency, treat 'bubble boy disease'? - San Francisco Business Times

Pioneering stem cell gene therapy cures infants with bubble baby disease – Medical Xpress

March 28, 2017 by Tiare Dunlap Evangelina Vaccaro (far right), who in 2012 received treatment developed by UCLAs Dr. Donald Kohn for bubble baby disease, with her family before her first day of school. Credit: Courtesy of the Vaccaro family

UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.

In a phase 2 clinical trial led by Dr. Donald Kohn of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohn's treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the child's own cells, has cured 30 out of 30 babies during the course of several clinical trials.

Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.

Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzymea lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipient's body rejecting the transplanted cells.

The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patient's own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each child's bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.

The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.

The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.

The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.

Explore further: Stem cell researcher pioneers gene therapy cure for children with "Bubble Baby" disease

More information: Clinical efficacy of gene-modified stem cells in adenosine deaminasedeficient immunodeficiency. http://www.jci.org/articles/view/90367

UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called "Bubble Baby" disease, a life-threatening ...

For the last several decades, scientists worldwide have been seeking to harness the power of stem cells to develop therapies for human diseases and conditions. At UCLA's Broad Stem Cell Research Center, the potential to bring ...

New research published online today in Blood, the Journal of the American Society of Hematology (ASH), reports that children with "bubble boy disease" who undergo gene therapy have fewer infections and hospitalizations than ...

Gene therapy can safely rebuild the immune systems of older children and young adults with X-linked severe combined immunodeficiency (SCID-X1), a rare inherited disorder that primarily affects males, scientists from the National ...

Researchers have found that gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited ...

Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy. A team of researchers ...

A new study published in Nature Communications and co-authored by Northwestern Medicine scientists shows how two proteins of the Ca2+ release-activated Ca2+ (CRAC) channel family interact with each other to control the flow ...

UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year ...

Wellcome Trust Sanger Institute scientists and their collaborators have developed a new analysis tool that was able to show, for the first time, which genes were expressed by individual cells in different genetic versions ...

The liver is crucial for the detoxification of the human body. The exposure to toxins makes it particularly prone to drug-induced injury. Cholestasis, the impairment of bile flow, is therefore a common problem of drug development ...

Men unable to have an erection after prostate surgery enjoyed normal intercourse thanks to stem cell therapy, scientists are to report Saturday at a medical conference in London.

McMaster University researchers have discovered that while survivors of childhood brain tumours have a similar Body Mass Index (BMI) to healthy children with no cancer, they have more fat tissue overall, and especially around ...

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Pioneering stem cell gene therapy cures infants with bubble baby disease - Medical Xpress

Regenxbio (RGNX) Names Olivier Danos, Ph.D as Chief Scientific Officer – StreetInsider.com

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REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced that Olivier Danos, Ph.D., is joining REGENXBIO in the newly created position of Chief Scientific Officer. Dr. Danos will report to Kenneth T. Mills, REGENXBIOs President and Chief Executive Officer.

Dr. Danos joins REGENXBIO from Biogen Inc., where he was Senior Vice President, Cell and Gene Therapy. At Biogen, Dr. Danos led company efforts dedicated to identifying and developing new technologies for gene transfer and genome engineering. Dr. Danos also co-founded and is an executive member of the board of directors of Lysogene, a NAV Technology Licensee focused on the development of gene therapy product candidates for the treatment of Mucopolysaccharidosis Type IIIA.

Olivier brings established industry leadership and scientific expertise in the development of gene therapies and the field of genome engineering to REGENXBIO, said Dr. James M. Wilson, M.D., Ph.D. REGENXBIOs scientific founder and director of the University of Pennsylvania (Penn) Gene Therapy Program. My relationship with Olivier can be traced back to the early stages of our careers, when he and I shared a bench at the Whitehead laboratories. He is an innovative scientist who has been at the forefront of the industry, and I look forward to collaborating with him on the advancement of scientific research at REGENXBIO.

We are excited to enhance the breadth and depth of our scientific research team. Olivier and Jim are two renowned gene therapy pioneers who have been key players in the success of the field, said Mr. Mills. We look forward to leveraging Oliviers rich industry experience and remarkable understanding of the space, including his work with the NAV Technology Platform, as we build a robust clinical pipeline of gene therapy product candidates with the goal of improving treatment options in many diseases.

The NAV Technology Platform has the potential to significantly alter the course of disease and deliver enhanced health outcomes to patients in need, said Dr. Danos. I am thrilled to have the opportunity to lead scientific research at REGENXBIO as we continue to advance this groundbreaking technology in a broad range of diseases that are not effectively addressed by existing drug classes.

Prior to Biogen, Dr. Danos served as Senior Vice President, Molecular Medicine, Synthetic Biology and Gene Regulation at Kadmon Pharmaceuticals. Earlier in his career, Dr. Danos was Director of the Gene Therapy Consortium of the University College of London, Scientific Director at Genethon and Senior Director of Research at Somatix Therapy Corporation. Dr. Danos has directed research focused on gene therapy at the Necker - Enfants Malades Hospital in Paris, the French National Centre for Scientific Research and the Pasteur Institute in Paris.

Dr. Danos received a Ph.D. in Biology at the University of Paris Diderot and the Pasteur Institute, and a Master in Science in Genetics and Mathematics from the University of Paris Orsay. Dr. Danos is a founding member of the European Society of Gene and Cell Therapy.

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Regenxbio (RGNX) Names Olivier Danos, Ph.D as Chief Scientific Officer - StreetInsider.com

Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder – Science Daily


Science Daily
Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder
Science Daily
In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene ...

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Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder - Science Daily

Money for research, hope for a cure: Race honors Eliza O’Neill and Sanfilippo kids – The State


The State
Money for research, hope for a cure: Race honors Eliza O'Neill and Sanfilippo kids
The State
On Saturday, Hickey and some of her genetic counseling peers joined dozens of others at a 5K race fundraiser to increase awareness of the rare, degenerative disease and raise money for research into an effective treatment or cure. There's a lot of ...
They isolated themselves for 726 days to give their daughter a chance at lifeDurham Herald Sun

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Money for research, hope for a cure: Race honors Eliza O'Neill and Sanfilippo kids - The State

Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder – Medical Xpress

March 23, 2017 This image shows stereocilia bundles on inner hair cells from whirler mice after whirlin gene therapy. These hair-like protrusions allow sensory hair cells to detect sound and motion. The whirler mutant mouse has very short stereocilia bundles. After whirlin gene therapy, the stereocilia bundles are increased to normal length (red) and whirlin expression is restored (green). Credit: Johns Hopkins Medicine

In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene therapy was able to restore balance and hearing in genetically modified mice that mimic Usher Syndrome, a genetic condition in humans characterized by partial or total hearing loss, dizziness, and vision loss that worsens over time. The hearing loss and dizziness is caused by abnormalities of the inner ear.

Dizziness and hearing loss are among the most common disabilities affecting humans and can be severe and debilitating. According to the National Health and Nutrition Examination Survey, more than 35% of U.S. adults aged 40 years and older have some degree of balance dysfunction, a major cause of falls in the elderly. According to the Centers for Disease Control, approximately one in three people in the United States between the ages of 65 and 74 has hearing loss, and nearly half of those older than 75 have difficulty hearing. Men are more likely to experience hearing loss than women.

Primary investigator Wade Chien, M.D., a neurootologist and associate professor with the Johns Hopkins Otolaryngology-Head and Neck Surgery team who also practices at the Johns Hopkins Healthcare and Surgery Center in Suburban Hospital in Bethesda, MD., and his team administered gene therapy to the inner ears of genetically modified mice carrying a mutation in a gene which is associated Usher syndrome. These mutant mice are deaf and have significant balance problems from birth. After gene therapy administration, the balance function of the mutant mice was completely restored. In addition, these mutant also had improvement in hearing. This study was one of the first to show that gene therapy can be used to improve hearing and balance functions in a mouse model of hereditary hearing loss. This study was funded by the NIDCD intramural research program.

"Inner ear gene therapy offers tremendous potential as a new way to help patients with hearing loss and dizziness," Chien said.

While the positive results are striking the researchers caution that the results are preliminary and will require additional research in humans to demonstrate fully their utility in treating humans. However, they are optimistic that their data indicate that inner ear gene therapy hold promise for treating a variety of human inherited vestibular and hearing disorders, including Usher syndrome.

Explore further: Number of people in US with hearing loss expected to nearly double in coming decades

More information: Kevin Isgrig et al. Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome, Molecular Therapy (2017). DOI: 10.1016/j.ymthe.2017.01.007

In a study published online by JAMA Otolaryngology-Head & Neck Surgery, Adele M. Goman, Ph.D., of Johns Hopkins University, Baltimore, Md., and colleagues used U.S. population projection estimates with current prevalence ...

In the summer of 2015, a team at Boston Children's Hospital and Harvard Medical School reported restoring rudimentary hearing in genetically deaf mice using gene therapy. Now the Boston Children's research team reports restoring ...

In a study published online by JAMA Otolaryngology-Head & Neck Surgery, Kathleen M. Schieffer, B.S., of the Pennsylvania State University College of Medicine, Hershey, Pa., and colleagues examined the association between ...

Severe hearing loss is the third most prevalent chronic condition in older Americans and more than 15% of people in their 30s are also affected. The condition leads to communication problems, social isolation, depression, ...

A new gene therapy approach can reverse hearing loss caused by a genetic defect in a mouse model of congenital deafness, according to a preclinical study published by Cell Press in the July 26 issue of the journal Neuron. ...

Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both.

Monash University researchers have discovered the mechanism underlying the fainting disorder, Postural Orthostatic Tachycardia Syndrome (POTS), the condition famously affecting the former lead singer of The Wiggles.

A person carrying variants of two particular genes could be almost three times more likely to develop multiple sclerosis, according to the latest findings from scientists at The University of Texas Medical Branch at Galveston ...

Researchers with the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and their collaborators, have successfully used facial recognition software to diagnose a rare, genetic disease ...

In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of ...

An international team of researchers from institutions around the world, including Baylor College of Medicine, has discovered that mutations of the OTUD6B gene result in a spectrum of physical and intellectual deficits. This ...

Researchers at Baylor College of Medicine, Texas Children's Hospital and Rice University have uncovered a gene mutation that may provide answers to unexplained female infertility. The study appears in Scientific Reports, ...

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Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder - Medical Xpress

Cancer Gene Therapy Market Deep Research Study with Forecast by 2025 – MilTech

Albany, NY (SBWIRE) 03/24/2017 Global Cancer Gene Therapy Market: Overview

Cancer results from the multiple mutations in a single cell that makes it to proliferate out of control. Cancer cells invade new cellular territories, have a high metabolic rate, and an altered shape. The various methods to treat cancers are surgery, radiation, and chemotherapy. When the aforementioned therapies fail to achieve desired results, gene therapy is leveraged. Gene therapy involves the insertion of a functional gene, also known as therapeutic DNA, into the cells of a cancer patient to rectify the metabolism, to change or repair an acquired genetic abnormality, and to provide a new function to a cell. The two main types of gene therapy are germinal and somatic.

Global Cancer Gene Therapy Market: Key Trends

Majorly promoting the global cancer gene therapy market is the swift pace of technological breakthroughs and the growing popularity of emerging genomic technologies like next-generation sequencing and high-density DNA microarrays. Additionally, the government support for these technologies is also slated to stoke growth in the near future. The Center for Disease Control and Prevention (CDC), for example, supports screening programs for breast cancer control and cervical and colorectal cancers among low-income group women sans health insurance in the U.S.

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Besides, the rising occurrence of cancer worldwide is will substantially drive up demand for gene therapy in the years ahead. According to WHO, cases of cancer will likely touch US$15 million mark by the end of the decade.

Global Cancer Gene Therapy Market: Market Potential

At present, most of the cancer gene therapy products are in being tested. The market is predicted to grow once the trials bear results. An US pharmaceutical company named Kite Pharma, for example, recently revealed the results from the initial six months of the trial of a new gene therapy treatment called CAR-T cell therapy. It helped up patients own immune cells and has eliminated the disease from one third of terminal patients. Around 36 per cent of the 101 patients on the trial were still in complete remission at six months, and eight in 10 saw their cancer reduced by at least half during the study.

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Groundbreaking therapies such as this is slated to revolutionize the global cancer gene therapy market.

At present, adenoviral vector is a popular oncology application because of its effective nuclear mechanism and low pathogenicity. Adenoviral vectors are leveraged in gene replacement approaches, suicide gene, gene-based immunotherapy, and syndicate gene with chemotherapy. Retroviral vector-mediated gene transfer also plays a key role in the gene therapy industry for it brings about the crucial benefit of changing the single stranded RNA genome into a double stranded DNA molecule, which eventually integrates into the target cell genome.

Global Cancer Gene Therapy Market: Regional Outlook

North America and Europe are key regions in the global cancer gene therapy market on account of a massive elderly population and significant technological progress in the region. In the years ahead, however, the market is Asia Pacific is forecasted to surge on account of supportive government initiatives, improving economy, bettering healthcare infrastructure, and growing thrust on research and development.

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Global Cancer Gene Therapy Market: Competitive Analysis

Some of the prominent players in the global cancer gene therapy market are Altor Bioscience Corporation, SiBiono., Shanghai Sunway Biotech company Limited, BioCancell, GlobeImmune, Inc.,Aduro Biotech, OncoGeneX, New Link Genetics., ZioPharm Oncology, and GENELUX. At present the market is led by small pioneering biotech firms who may eventually collaborate with prominent players for clinical development or commercialization of products.

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Cancer Gene Therapy Market Deep Research Study with Forecast by 2025 - MilTech

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