Urocortin 3 gene therapy increases systolic and diastolic function in heart failure
(Mary Ann Liebert, Inc./Genetic Engineering News) Mice with heart failure that were treated with AAV8-based gene therapy to deliver the protein urocortin 3 (UCn3) had increased blood levels of UCn3 over a 5-week period and improved heart function. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - November 14, 2018 Category: Biology Source Type: news

Mesothelioma Patients Can Benefit from Genetic Testing
All patients diagnosed with malignant mesothelioma should undergo genetic testing to better identify the most appropriate treatment plan, according to Dr. Michele Carbone, the director of thoracic oncology at the University of Hawaii Cancer Center. Genetic testing also could lead to a more accurate prognosis and help family members determine if they are more susceptible to the cancer. Carbone estimates only one-third of all mesothelioma patients today have undergone the testing. “Some of these patients could definitely benefit from genetic testing,” Carbone told The Mesothelioma Center at Asbestos.com. Carbon...
Source: Asbestos and Mesothelioma News - November 13, 2018 Category: Environmental Health Authors: Matt Mauney Source Type: news

Novartis' $4 Million Gene Therapy -- Real Price Or A Negotiation Ploy?
We are in a wondrous age of medicine where CURES for certain types of cancer, rare diseases, blindness and other scourges are imminent. We now need to figure out how to pay for them. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 13, 2018 Category: Pharmaceuticals Authors: John LaMattina, Contributor Tags: NASDAQ:AVXS NYSE:NVS Source Type: news

Combination gene therapy more effective in cartilage preservation in osteoarthritis
(Mary Ann Liebert, Inc./Genetic Engineering News) A combinatorial gene therapy approach -- one designed to inhibit inflammation and one targeting protection against cartilage degeneration -- was shown to preserve articular carti-lage better than each approach alone in animal models of both moderate and severe post-traumatic osteoarthritis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 13, 2018 Category: International Medicine & Public Health Source Type: news

New gene therapy reprograms brain glial cells into neurons
(Penn State) A new gene therapy can turn certain brain glial cells into functioning neurons, which in turn could help repair the brain after a stroke or during neurological disorders like Alzheimer's or Parkinson's diseases. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - November 5, 2018 Category: Biology Source Type: news

TGen, City of Hope scouring metro Phoenix to build cell therapy manufacturing space
Shortage of cell and gene therapy products creates need for Phoenix manufacturing facility (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - November 2, 2018 Category: American Health Authors: Angela Gonzales Source Type: news

TGen, City of Hope scouring metro Phoenix to build cell therapy manufacturing space
Shortage of cell and gene therapy products creates need for Phoenix manufacturing facility (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - November 2, 2018 Category: Biotechnology Authors: Angela Gonzales Source Type: news

Why Pennsylvania's life sciences community is a wise investment (and why we need more of it)
Earlier this month, Philadelphia ’s Spark Therapeutics announced that it will be moving into the Schuylkill Yards development in University City, signaling an expansion of its research and development operations that is expected to create 500 new jobs. Spark Therapeutics, a commercial gene therapy company, represents one of m any cutting-edge life science companies birthed right here in Philadelphia. Their story, along with countless others, will undoubtedly be the talk of Life Sciences Future,… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 29, 2018 Category: Biotechnology Authors: Daniel K. Fitzpatrick and Laura Fogerty Source Type: news

Why Pennsylvania's life sciences community is a wise investment (and why we need more of it)
Earlier this month, Philadelphia ’s Spark Therapeutics announced that it will be moving into the Schuylkill Yards development in University City, signaling an expansion of its research and development operations that is expected to create 500 new jobs. Spark Therapeutics, a commercial gene therapy company, represents one of m any cutting-edge life science companies birthed right here in Philadelphia. Their story, along with countless others, will undoubtedly be the talk of Life Sciences Future,… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - October 29, 2018 Category: American Health Authors: Daniel K. Fitzpatrick and Laura Fogerty Source Type: news

Fewer Injections With New Wet Macular Degeneration Therapies Fewer Injections With New Wet Macular Degeneration Therapies
New treatments in development — including gene therapy — will likely mean fewer injections for patients with neovascular age-related macular degeneration, studies indicate.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - October 28, 2018 Category: Consumer Health News Tags: Ophthalmology News Source Type: news

GenSight Biologics launches trial for gene therapy, eye device combo
GenSight Biologics (EPA:SIGHT) said today that the first person was treated in a first-in-man trial evaluating GenSight’s gene therapy candidate, GS030-DP, in combination with a wearable optronic visual stimulation device, GS030-MD. Scientists are evaluating the drug-device combination in 18 people with retinitis pigmentosa. Get the full story at our sister site, Drug Delivery Business News. The post GenSight Biologics launches trial for gene therapy, eye device combo appeared first on MassDevice. (Source: Mass Device)
Source: Mass Device - October 26, 2018 Category: Medical Devices Authors: Sarah Faulkner Tags: Clinical Trials Drug-Device Combinations Optical/Ophthalmic Pharmaceuticals Wall Street Beat gensightbiologics Source Type: news

A Brutal Clock
Amber Olsen is racing against time to fund a gene therapy that might save her dying 5-year-old from a rare genetic disease. (Source: Science - The Huffington Post)
Source: Science - The Huffington Post - October 26, 2018 Category: Science Source Type: news

Gene Therapy for Parkinson's Symptoms Is Promising
A new treatment for Parkinson’s disease uses a virus to deliver gene therapy to a targeted area of the brain. While not a cure, scientists hope it will help control symptoms of the progressive neurological disease. (Source: WebMD Health)
Source: WebMD Health - October 25, 2018 Category: Consumer Health News Source Type: news

Gene Therapy for Parkinson's Symptoms Shows Promise
THURSDAY, Oct. 25, 2018 -- A new gene therapy might help improve motor symptoms in people with Parkinson's disease who aren't responding to other therapies, an early study has found. " This is not a cure of Parkinson's disease, " said James Beck,... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - October 25, 2018 Category: General Medicine Source Type: news

Parkinson's Gene Therapy Shows Signs of Motor Improvement
(MedPage Today) -- Early open-label trial in advancing Parkinson's disease showed treatment was safe, well-tolerated (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - October 24, 2018 Category: Neurology Source Type: news

Innovative gene therapy trial for Parkinson's disease
(University College London) A trial for a new gene therapy, known as AXO-Lenti-PD, aimed at improving the supply of dopamine in the brains of people with Parkinson's disease has been launched by researchers at UCL and University College London Hospitals (UCLH). (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - October 24, 2018 Category: Biology Source Type: news

Gene Therapy Shows Early Signs of Parkinson's Improvement Gene Therapy Shows Early Signs of Parkinson's Improvement
This MRI-guided approach transfers the gene therapy directly to specific regions of the brain where conversion to dopamine is impaired.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - October 23, 2018 Category: Consumer Health News Tags: Neurology & Neurosurgery News Source Type: news

CavoGene LifeSciences Licenses Novel Gene Therapy for CNS Disorders
Agreement with University California San Diego Provides License for Production of SynCav1, an Investigational Novel Gene Therapy for Treatment of Alzheimer's Disease and Amyotrophic Lateral Sclerosis GLEN BURNIE, Md., Oct. 23, 2018 -- (Healthcare Sales ... Biopharmaceuticals, Neurology, Licensing CavoGene LifeSciences, SynCav1, Alzheimer's disease, neurodegenerative disease (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - October 23, 2018 Category: Pharmaceuticals Source Type: news

Boston biopharma commercializing Nationwide Children's gene therapies to add Columbus office, 100 jobs
Sarepta Therapeutics Inc. plans to open the first phase of its Gene Therapy Center of Excellence in Columbus by spring and expand throughout 2019 – eventually 85,000 square feet and 100 high-paying jobs. The CEO thinks Columbus could be for gene therapy what Cambridge is for biotech. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - October 16, 2018 Category: American Health Authors: Carrie Ghose Source Type: news

Boston biopharma commercializing Nationwide Children's gene therapies to add Columbus office, 100 jobs
Sarepta Therapeutics Inc. plans to open the first phase of its Gene Therapy Center of Excellence in Columbus by spring and expand throughout 2019 – eventually 85,000 square feet and 100 high-paying jobs. The CEO thinks Columbus could be for gene therapy what Cambridge is for biotech. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 16, 2018 Category: Biotechnology Authors: Carrie Ghose Source Type: news

How OHSU restored a 4-year-old's sight (Photos)
Until a month ago, 4-year-old Caspian Soto had to use a headlamp and cane to navigate the world around him. He couldn ’t see the stars in the sky, or anything much in dim light. The Portland boy was born with a rare genetic mutation that causes Leber’s congenital amaurosis, a condition that made his vision dark and blurry. That all changed for Caspian when he underwent a new gene therapy treatment at the OHSU Casey Eye Institute called Luxturna. Doctors injected a modified virus into Caspian’s… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 16, 2018 Category: Pharmaceuticals Authors: Elizabeth Hayes Source Type: news

This RNA-based technique could make gene therapy more effective
(Massachusetts Institute of Technology) MIT biological engineers have devised a way to regulate the expression of messenger RNA once it gets into cells, giving them more precise control over gene therapy treatments for cancer and other diseases. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - October 16, 2018 Category: Cancer & Oncology Source Type: news

Multidisciplinary team to develop stem cell-based approaches to restore vision
(University of Pennsylvania) Gene therapies have had success in treating blindness but can't save areas of the retina where cells have already died. In a new effort, University of Pennsylvania scientists John Wolfe, also of CHOP, and William Beltran, along with David Gamm of the University of Wisconsin-Madison, will attempt to develop a stem-cell-based approach that restores vision. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - October 16, 2018 Category: International Medicine & Public Health Source Type: news

Sarepta CEO charts path forward on Duchenne drug amid stock slide
Sarepta has seen its share price decline in the wake of data from a small study of its gene therapy drug for Duchenne muscular dystrophy. But the Cambridge biotech is moving forward with its clinical trial work and expansion plans. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 11, 2018 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

Spark Therapeutics to add up to 500 jobs in West Philadelphia expansion
Spark Therapeutics Inc. said Thursday it is expanding its research-and-development operations in West Philadelphia in a move expected to create nearly 500 high-paying jobs. The gene therapy company spun out of Children's Hospital of Philadelphia is getting a $2 million Department of Community and Economic Development grant and a $7.5 million grant from the Redevelopment Assistance Capital Program to support the project. Spark's new site will be located on Market Str eet in Drexel University’s… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - October 11, 2018 Category: American Health Authors: John George Source Type: news

Meet the $172.5 million biotech IPO from the U.K. with big plans to grow in the Bay Area
Orchard Rx Ltd., a London-based gene therapy company targeting "bubble boy" disease and other rare genetic conditions, wants to raise $172.5 million through an initial public offering that in part will bankroll a key Bay Area expansion. Orchard, whose shareholders include drug giant GlaxoSmithKline plc (NYSE: GSK), is the latest biotech company to take advantage of Wall Street's open cash window. It filed its IPO plans Thursday, less than two months after raising $148 million in an oversubscribed… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 8, 2018 Category: Biotechnology Authors: Ron Leuty Source Type: news

Meet the $172.5 million biotech IPO from the U.K. with big plans to grow in the Bay Area
Orchard Rx Ltd., a London-based gene therapy company targeting "bubble boy" disease and other rare genetic conditions, wants to raise $172.5 million through an initial public offering that in part will bankroll a key Bay Area expansion. Orchard, whose shareholders include drug giant GlaxoSmithKline plc (NYSE: GSK), is the latest biotech company to take advantage of Wall Street's open cash window. It filed its IPO plans Thursday, less than two months after raising $148 million in an oversubscribed… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - October 5, 2018 Category: Health Management Authors: Ron Leuty Source Type: news

Meet the $172.5 million biotech IPO from the U.K. with big plans to grow in the Bay Area
Orchard Rx Ltd., a London-based gene therapy company targeting "bubble boy" disease and other rare genetic conditions, wants to raise $172.5 million through an initial public offering that in part will bankroll a key Bay Area expansion. Orchard, whose shareholders include drug giant GlaxoSmithKline plc (NYSE: GSK), is the latest biotech company to take advantage of Wall Street's open cash window. It filed its IPO plans Thursday, less than two months after raising $148 million in an oversubscribed… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - October 5, 2018 Category: Biotechnology Authors: Ron Leuty Source Type: news

Sarepta Gene Therapy Appears To Help Fourth Boy With Duchenne Muscular Dystrophy
“The cautionary note is this all has to be confirmed in a larger trial,” says Douglas Ingram, Sarepta’s president and chief executive. “Our goal is to bring this therapy rapidly to patients around the world,” Ingram says. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - October 3, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Source Type: news

CAR T-Cell Therapy May ‘ Change the Paradigm of Treating Mesothelioma ’
Thoracic surgeon and scientist Dr. Prasad Adusumilli at Memorial Sloan Kettering (MSK) Cancer Center believes customized gene therapy will soon change the way pleural mesothelioma is treated. Adusumilli, director of the Mesothelioma Program at MSK, has worked for a decade on developing tumor immunology for thoracic malignancies. He is currently the principal investigator of a phase I clinical trial involving specially prepared immune cells — chimeric antigen receptor (CAR) T cells — that have shown impressive efficacy with mesothelioma. “I think this is going to change the paradigm of treating mesotheliom...
Source: Asbestos and Mesothelioma News - October 1, 2018 Category: Environmental Health Authors: Matt Mauney Source Type: news

International Symposium on Mesothelioma to Focus on Immunotherapy
The eighth annual International Symposium on Malignant Pleural Mesothelioma Saturday at UCLA will reflect the rapidly evolving philosophy on future treatment of this rare cancer. Change is coming. The Symposium, which was first held in 2011 to help bring awareness to lung-sparing surgery, will highlight groundbreaking discoveries and the latest advances in immunotherapy and gene therapy — believed to be the future of cancer care. The longtime chemotherapy and surgery standard-of-care routine is fading. The symposium at the Luskin Conference Center on the UCLA campus in Los Angeles will help explain why. “There ...
Source: Asbestos and Mesothelioma News - September 26, 2018 Category: Environmental Health Authors: Matt Mauney Source Type: news

Mitochondrial diseases could be treated with gene therapy
Researchers have developed a genome editing tool for the potential treatment of mitochondrial diseases. The study applied an experimental gene therapy treatment in mice and successfully targeted and eliminated the damaged DNA in mitochondria. (Source: Medical Research Council General News)
Source: Medical Research Council General News - September 25, 2018 Category: Research Source Type: news

Gene therapy may be effective against mitochondrial diseases
A newly developed gene editing tool can potentially treat mitochondrial diseases, which occur when the energy-producing structures malfunction. (Source: Health News - UPI.com)
Source: Health News - UPI.com - September 24, 2018 Category: Consumer Health News Source Type: news

Nationwide Children's gene therapy spinoff acquired for $100M
Yet another gene therapy spinoff from Nationwide Children's Hospital has been acquired for $100 million. Celenex is developing a treatment for Batten Disease, a rare, fatal neurological condition that causes blindness and progressive loss of speech and movement. Children who inherit it typically don't live past age 12. The startup was acquired by Amicus Therapeutics Inc., a Cranbury, New Jersey-base d biotechnology company that focuses on rare metabolic disorders. Columbus-based Rev1 Ventures,… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - September 21, 2018 Category: Pharmaceuticals Authors: Carrie Ghose Source Type: news

Nationwide Children's gene therapy spinoff acquired for $100M
Yet another gene therapy spinoff from Nationwide Children's Hospital has been acquired for $100 million. Celenex is developing a treatment for Batten Disease, a rare, fatal neurological condition that causes blindness and progressive loss of speech and movement. Children who inherit it typically don't live past age 12. The startup was acquired by Amicus Therapeutics Inc., a Cranbury, New Jersey-base d biotechnology company that focuses on rare metabolic disorders. Columbus-based Rev1 Ventures,… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 21, 2018 Category: Biotechnology Authors: Carrie Ghose Source Type: news

European advisory panel recommends approval of Spark's gene therapy
The European Medicines Agency advisory panel voted Friday to recommend approval of Luxturna, a one-time gene therapy developed by Philadelphia-based Spark Therapeutics for an inherited eye disorder that leads to blindness if untreated. Luxturna received Food and Drug Administration approval in late 2017. The European Medicines Agency typically follows the removal of its advisory panels. If the treatment is approved in Europe, it would become the first gene therapy for a genetic disease approved… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 21, 2018 Category: Biotechnology Authors: John George Source Type: news

Amicus bets on gene therapy with Celenex buy
NJ-based Amicus agreed to pay $100 million up front to buy privately-held Celenex, giving Amicus a portfolio of 10 experimental gene therapies. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - September 21, 2018 Category: Pharmaceuticals Source Type: news

Gene therapy in mouse models of deafness and balance dysfunction - Wang L, Kempton JB, Brigande JV.
Therapeutic strategies to restore hearing and balance in mouse models of inner ear disease aim to rescue sensory function by gene replacement, augmentation, knock down or knock out. Modalities to achieve therapeutic effects have utilized virus-mediated tra... (Source: SafetyLit)
Source: SafetyLit - September 19, 2018 Category: International Medicine & Public Health Tags: Sensing and Response Issues Source Type: news

Bascom Palmer treats first US patient in Nightstar gene therapy
(University of Miami Miller School of Medicine) A Puerto Rican patient with X-linked retinitis pigmentosa (XLRP) is hoping to save his vision after an innovative gene therapy procedure at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine. On August 23, Julio Adorno Nieves, 23, became the first US patient to be given new genes for his inherited blinding condition in a worldwide Nightstar Therapeutics clinical trial. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 19, 2018 Category: International Medicine & Public Health Source Type: news

Is the end of the recombinant DNA Advisory Committee (RAC) a good thing?
(Mary Ann Liebert, Inc./Genetic Engineering News) Recently, the U.S. National Institutes of Health (NIH) and Food and Drug Administration (FDA) called for the eliminating involvement of the Recombinant DNA Advisory Committee (RAC) in human gene therapy experiments, marking the end of an era of federal government oversight. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - September 19, 2018 Category: Biology Source Type: news

Biotech co. hosts career fair at new Pearland facility, aims to hire 200 by end of 2019
Switzerland-based biotech giant Lonza Group Ltd. is moving forward with plans to double its workforce at its recently opened Pearland facility, billed as the largest dedicated cell and gene therapy manufacturing facility in the world. The 300,000-square-foot facility officially opened in April with 170 employees. Ricardo Jimenez, site director at Lonza Houston Inc., told the Houston Business Journal at the time that the space could support more than 400 workers.  Now, the company is holding its… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - September 18, 2018 Category: Biotechnology Authors: Olivia Pulsinelli Source Type: news

Biotech co. hosts career fair at new Pearland facility, aims to hire 200 by end of 2019
Switzerland-based biotech giant Lonza Group Ltd. is moving forward with plans to double its workforce at its recently opened Pearland facility, billed as the largest dedicated cell and gene therapy manufacturing facility in the world. The 300,000-square-foot facility officially opened in April with 170 employees. Ricardo Jimenez, site director at Lonza Houston Inc., told the Houston Business Journal at the time that the space could support more than 400 workers.  Now, the company is holding its… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - September 18, 2018 Category: American Health Authors: Olivia Pulsinelli Source Type: news

Cure for cocaine addiction in reach, say scientists
Gene therapists have developed a stem cell implant that could help overcome addiction and prevent overdosesA radical gene therapy for drug addiction has been shown to dampen down cravings for cocaine and protect against overdoses of the substance that would normally be lethal.The therapy uses implants of stem cells which have been genetically engineered to release a powerful enzyme that removes the class A drug from the bloodstream.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - September 17, 2018 Category: Science Authors: Ian Sample Science editor Tags: Science Drugs Drugs trade Genetics Society World news Medical research Stem cells Biology Source Type: news

Counting (on) sheep? Promising gene therapy for visually impaired sheep now safe for human trials
(The Hebrew University of Jerusalem) A promising gene therapy for visually impaired sheep is now safe for human trials. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - September 17, 2018 Category: International Medicine & Public Health Source Type: news

Surgery for Mesothelioma No Longer Recommended in UK
Aggressive surgery for the treatment of malignant pleural mesothelioma (MPM) — unless part of a clinical trial — is no longer being recommended in the United Kingdom. The British Thoracic Society recently published its Mesothelioma Management Guidelines for 2018 and removed surgery from its recommendations. The belief was that potential harm was not worth the risk. The backtrack on surgery included the extrapleural pneumonectomy (EPP), the extended pleurectomy and decortication (P/D) and the partial pleurectomy (PP) — all of which are being done at cancer centers in the United States. “Surgery is a ...
Source: Asbestos and Mesothelioma News - September 11, 2018 Category: Environmental Health Authors: Matt Mauney Source Type: news

Electron microscopy provides new view of tiny virus with therapeutic potential
(Salk Institute) Researchers from the Salk Institute and the University of Florida are reporting how they used cryo-EM to show the structure of a version of a virus called an AAV2, advancing the technique's capabilities and the virus' potential as a delivery vehicle for gene therapies. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - September 7, 2018 Category: Biology Source Type: news

NIH Vision Researcher Among Seven to Receive Top Global Award NIH Vision Researcher Among Seven to Receive Top Global Award
T. Michael Redmond and six others were honored for developing gene therapy for the blinding disorder Leber congenital amaurosis. It is the only pharmacologic treatment for an inherited retinal disease.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - September 5, 2018 Category: Consumer Health News Tags: Ophthalmology News Source Type: news

Deloitte, Vineti partner for cloud-based precision medicine platform to speed innovation
Deloitte's ConvergeHEALTH will integrate tech with Vineti, the gene therapy initiative co-developed by GE and the Mayo Clinic. (Source: mobihealthnews)
Source: mobihealthnews - September 5, 2018 Category: Information Technology Source Type: news

Early Results Give Hope For Human Gene Editing to Change DNA
(PHOENIX) — Early, partial results from a historic gene editing study give encouraging signs that the treatment may be safe and having at least some of its hoped-for effect, but it’s too soon to know whether it ultimately will succeed. The results announced Wednesday are from the first human test of gene editing in the body, an attempt to permanently change someone’s DNA to cure a disease — in this case, a genetic disorder called Hunter syndrome that often kills people in their teens. In two patients who got a medium dose of the treatment, urine levels of large sugar compounds that are hallmarks of ...
Source: TIME: Health - September 5, 2018 Category: Consumer Health News Authors: MARILYNN MARCHIONE / AP Tags: Uncategorized Disease onetime Source Type: news

Gene therapy breakthrough wins world's largest vision award
Seven scientists in the United States and Britain who have come up with a revolutionary gene therapy cure for a rare genetic form of childhood blindness won a 1 million euro ($1.15 million)prize on Tuesday, Portugal's Champalimaud Foundation said. (Source: Reuters: Health)
Source: Reuters: Health - September 4, 2018 Category: Consumer Health News Tags: healthNews Source Type: news