Abstract
Muscular dystrophies are progressive muscle-wasting diseases often caused by defects of genes encoding sarcolemmal or sarcolemma-associated proteins and their consequential deficiency in muscle. For the muscular dystrophies, gene therapy has been widely studied, and several strategies involving the use of viral vector-mediated delivery have been developed to restore partial expression of deficient proteins. However, systemic, specific and safe delivery of therapeutic genes to skeletal muscles remains major hurdles for the clinical application of those strategies.
In this review, we first introduce genetic backgrounds and pathogenesis of muscular dystrophies and then some strategies of gene therapy, undertaken for potential treatment of muscular dystrophies. Furthermore, we describe and discuss current chemical and biological methods to carry therapeutic materials including oligonucleotides and cDNAs into individual muscle fibers and to deliver them to the musculature of the whole body.
