immune response to gene therapy in DMD

MDA-supported investigators found that intramuscular injections of microdystrophin genes improved muscle health in dystrophin-deficient dogs, a response not previously seen in large animals or humans

posted on January 15, 2013 - 2:34pm
For the first time, gene therapy using a highly miniaturized dystrophin gene resulted in significant improvement in muscle structure and function in dogs with a disorder mimicking human Duchenne muscular dystrophy.

Gene therapy researcher and MDA grantee Jerry Mendell discusses autoimmunity to dystrophin protein in Duchenne muscular dystrophy

posted on January 13, 2011 - 3:57pm
A January 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, probes a subject that's been on the minds of many researchers, doctors and families: autoimmunity (self-immunity) in Duchenne muscular dystrophy (DMD).