The long-term safety of gene transfer to treat neovascular age-related macular degeneration (NVAMD), and the production of two therapeutic proteins encoded by those genes for at least 2.5 years in the eyes of patients with ...
Oct 20, 2016
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1
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 ...
Sep 6, 2016
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76
"When you hear hoofbeats, think horses, not zebras," beginning med students learn. Ultrarare diseases are more like unicorns.
Aug 22, 2016
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5
As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist-oncologist ...
May 26, 2016
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1
Adolescents and young adults with a severe inherited immunodeficiency disorder improved following treatment with novel gene therapy developed at St. Jude Children's Research Hospital and at the National Institute of Allergy ...
Apr 20, 2016
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81
Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and ...
Jan 20, 2016
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35
Results from a long-term clinical trial conducted by cancer researchers at Houston Methodist Hospital show that combining radiation treatment with "suicide gene therapy," a technique in which prostate cancer cells are genetically ...
Dec 12, 2015
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467
Valerie Byers had long suspected that her son Will's diagnosis of autism was wrong. So when she saw a clip on the homepage of the Today Show about a little girl named Eliza, in late February, she knew instantly that 5-year-old ...
Jul 17, 2015
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27
A therapy that replaces the faulty gene responsible for cystic fibrosis in patients' lungs has produced encouraging results in a major UK trial.
Jul 3, 2015
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200
University of North Carolina (UNC) School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN).
Jun 25, 2015
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43
