An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...
With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...
UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...
Mayo Clinic researchers used milk-based nanoparticles to deliver siRNA into bile duct cancer cells. This targeted therapy ...
Nemours Children's Hospital is now offering Lyfgenia gene therapy. The treatment aims to reduce pain crises and hospital visits for young patients.
The current path to CAR-T cell therapy is, by any measure, a logistical ordeal. A patient’s immune cells must be drawn out of ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy ...
Sarepta Therapeutics’ report of a second fatality this year in a patient dosed with its Duchenne muscular dystrophy gene therapy raised the prospect it might have to pull the product from the market.
Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic lateral sclerosis (ALS), offering new hope for treating the devastating ...
The first-in-human Phase-I clinical trial for gene therapy of Hemophilia A has shown clinically significant outcomes.
From an early age, Allison Keeler ’05 (biotechnology) always knew she wanted to be a scientist. As an adult, that dream has come true as she is an assistant professor in the Department of Pediatrics ...
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