Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.

ABSTRACT: Pharmacologic strategies have provided modest improvement in the devastating muscle-wasting disease, Duchenne muscular dystrophy (DMD). Pre-clinical gene therapy studies have shown promise in the mdx mouse model; however, studies conducted after disease onset fall short of fully correcting muscle strength or protecting against contraction-induced injury. Here we examine the treatment effect on muscle physiology in aged dystrophic mice with significant disease pathology by combining two promising therapies: micro-dystrophin gene replacement and muscle enhancement with follistatin, a potent myostatin inhibitor. Individual treatments with micro-dystrophin and follistatin demonstrated marked improvement in mdx mice but were insufficient to fully restore muscle strength and response to injury t

SUBMITTER: Rodino-Klapac LR 

PROVIDER: S-EPMC3895965 | biostudies-literature | 2013 Dec

REPOSITORIES: biostudies-literature