Article
AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2013
Heller Kristin N, Montgomery Chrystal L, Janssen Paul Ml, Clark K Reed, Mendell Jerry R, Rodino-Klapac Louise R
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a severe muscle disease caused by mutations in the DMD gene, with loss of its gene product, dystrophin. Dystrophin helps link integral membrane proteins to the actin cytoskeleton and stabilizes the sarcolemma during muscle activity. We investigated an alternat...
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