gene therapy

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CRISPR enhances gene therapy to fight inherited diseases

Gene therapy has shown promise in treating inherited genetic diseases, but a major issue that has frustrated scientists remains: Replacing a “bad” gene with a healthy one often is a short-lived fix. Typically, the healthy replacement gene works for just a few weeks. Now, scientists at Washington University School of Medicine in St. Louis have

April 3rd, 2018|Categories: Around The State, General News|Tags: , , |

BCL11A-based gene therapy for sickle cell disease passes key preclinical test

September 06, 2016 Decades-old discovery about fetal hemoglobin is on track for clinical trial in the coming year A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center report today in

Gene Therapy Treats All Muscles in the Body in Muscular Dystrophy Dogs; Human Clinical Trials Are Next Step

Oct. 22, 2015 Story Contact: Christian Basi COLUMBIA, Mo. ­— Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy

Stem cell clinical trials are multiplying

By Bradley J. Fikes | 5:02 p.m. Oct. 3, 2015 It’s been a long wait for patients hoping for stem cell science to be turned into new treatments. After years of frustration at the incremental progress, stem cell therapies at last are streaming into clinical trials -- in San Diego, nationwide and across the world. In

Degenerating Neurons Respond to Gene Therapy Treatment for Alzheimer’s Disease

Postmortem brain studies suggest nerve growth factor safely triggered functional cell growth August 27, 2015 | By Scott LaFee Degenerating neurons in patients with Alzheimer’s disease (AD) measurably responded to an experimental gene therapy in which nerve growth factor (NGF) was injected into their brains, report researchers at University of California, San Diego School of

OHSU scientists unlock first critical step toward gene therapy treatment for patients with mitochondrial disease

07/15/15  Portland, Ore. Gene-based cures for human diseases are now on the horizon A study led by Shoukhrat Mitalipov, Ph.D., and Hong Ma, M.D., Ph.D., at the Center for Embryonic Cell and Gene Therapy at Oregon Health & Science University and the Oregon National Primate Research Center has revealed the first critical step in developing novel

Is Boston Children’s Hospital on its way to curing genetic deafness?

Jul 8, 2015, 1:08pm EDT Jessica Bartlett Boston Business Journal COURTESY/ POCKAFWYE VIA CREATIVE COMMONS ​Researchers used gene therapy to restore hearing in mice with a genetic form of… more Researchers at Boston Children’s Hospital and Harvard Medical School have used gene therapy to restore hearing in mice with a genetic form of deafness. The

Gene therapy for cystic fibrosis shows encouraging results

July 3, 2015 A therapy that replaces the faulty gene responsible for cystic fibrosis in patients' lungs has produced encouraging results in a major UK trial. The study was carried out by the UK Cystic Fibrosis Gene Therapy Consortium, a group of scientists and clinical teams from Imperial College London, the Universities of Oxford and

Stem cell gene therapy developed at UCLA holds promise for eliminating HIV infection

Method modifies immune cells to attack the virus, could be used to treat other diseases Mirabai Vogt-James | June 30, 2015 UCLA Broad Stem Cell Research CenterThe scientists, led by Jerome Zack (left) and Scott Kitchen, found that the technique decreased HIV levels in mice by 80 to 95 percent. Scientists at the UCLA Eli and

NIH-funded study points way forward for retinal disease gene therapy

Monday, May 4, 2015 Benefits for Leber congenital amaurosis peak after one to three years, then diminish Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients’ eyesight and the sensitivity of the retina within weeks of treatment. Both of these benefits, however, peaked one to