Gene therapy for muscular dystrophy - a review of promising progress

Abstract

This review considers the most significant progress in developing genetic therapeutic interventions for Duchenne muscular dystrophy (DMD), in order to illustrate the challenges facing gene therapy of all the muscular dystrophies (MDs). While in vivo repair of genetic mutations affecting muscle remains inefficient, the most promising interventions focus on supplementation of minimally sized transgenes encoding the abnormal muscle proteins. Intramuscular injection of recombinant viral vectors carrying therapeutic transgenes has yielded the most impressive amelioration of dystrophic muscle pathology in animal models, although achieving lasting, body-wide transgene delivery in the absence of a s..