Gene therapy for Duchenne muscular dystrophy

Gene therapy has great potential to treat Duchenne muscular dystrophy. Among many proposed strategies to deliver a therapeutic gene to muscle, recombinant adeno-associated virus-mediated gene transfer is the most promising. The recent isolation of new adeno-associated virus serotypes from human and nonhuman primates provides the opportunity to develop vectors that can achieve the long-term expression of a therapeutic gene in muscles of the entire body without detrimental effects. To translate the results from small animal models to clinical trials in humans, further work using larger animal models, such as dystrophic dogs or nonhuman primates, is required. This review also discusses recent progress in other gene transfer-related therapeutic approaches, including targeted exon skipping and gene correction.