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

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.

Keywords: cell therapy; gene therapy; genome editing; mRNA modifications; muscle regeneration; muscular dystrophy; stem cells.