Gene therapy leads to robust improvements in animal model of fatal muscle disease (University of Washington)

(Source: University of Washington) January 22, 2014 Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease know as X-linked myotubular myopathy. The findings, appearing in the January 22, 2014 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for this devastating disease. Watch a video by Brian Donohue on this study. Researchers at the  University of Washington, Généthon  in France, Boston Children's Hospital, and Virginia Polytechnic Institute and State University in Blacksburg, Va., conducted the study. The study was based on...