Therapy for neuromuscular disorders

Abstract

Research into therapeutic approaches for both recessive and dominant neuromuscular disorders has made great progress over the past few years. In the field of gene therapy, antisense-mediated exon skipping is being applied to bypass deleterious mutations in the dystrophin gene and restore dystrophin expression in animal models of muscular dystrophy. Approaches for the dominant genetic muscle diseases have turned toward elimination of the mutant gene product with anti-sense oligonucleotide therapy and RNA interference techniques. Refinements of adeno-associated viral vectors and strategies for their delivery are also leading towards future clinical trials. The discovery of new, multipotent cell lineages, some of which possess the ability to successfully engraft muscle following vascular delivery, presents exciting prospects for the field of stem cell therapy. These discoveries represent steady progress towards the development of effective therapies for a wide range of neuromuscular disorders.