Novel therapies for Duchenne muscular dystrophy

Abstract

The development of therapeutic strategies that overcome the unique problems posed by Duchenne muscular dystrophy (DMD) has lead to the development of many contemporary approaches to human disease in general. Various treatment approaches have been exploredas pharmacological therapies and cell-based, cytokine, and genetic therapies - that are all targeted to specific features of dystrophic DMD muscle pathology. In genetic therapies, the large size of the dystrophin gene has necessitated the development and use of novel functional minidystrophin and microdystrophin genes, muscle-specific promoter systems, and gutted adenoviral systems. In addition to these well defined viral strategies, plasmid..