Adeno-Associated Virus Vector-Mediated Minidystrophin Gene Therapy Improves Dystrophic Muscle Contractile Function in <i>mdx</i> Mice

Abstract excerpt

Duchenne muscular dystrophy (DMD) is the most common disabling and lethal genetic muscle disorder, afflicting 1 of every 3500 males. Patients with DMD experience progressive muscle degeneration and weakness and succumb to respiratory or cardiac failure by their early twenties. No treatment is currently available for DMD. Mutations in the dystrophin gene result in lack of a functional dystrophin protein in...

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