Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies

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Abstract

Muscular dystrophies are genetic disorders characterized by skeletal muscle wasting and weakness. Although there is no effective therapy, a number of experimental strategies have been developed over recent years and some of them are undergoing clinical investigation. In this review, we highlight recent developments and key challenges for strategies based upon gene replacement and gene/expression repair, including exon-skipping, vector-mediated gene therapy and cell therapy. Therapeutic strategies for different forms of muscular dystrophy are discussed, with an emphasis on Duchenne muscular dystrophy, given the severity and the relatively advanced status of clinical studies for this disease.

Muscular dystrophies are severe genetic disorders characterized by muscle wasting. Although there is no effective therapy, different experimental strategies have been developed. Here, we highlight recent experimental therapies based upon gene replacement and gene/expression repair, including exon-skipping, vector-mediated gene therapy and cell therapy. Different forms of muscular dystrophy will be discussed, with an emphasis on Duchenne muscular dystrophy.

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