GENE-THERAPY PROSPECTS FOR DUCHENNE MUSCULAR-DYSTROPHY

Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disor der caused by mutations in the dystrophin gene. The lack of adequate t herapy for this disease provides impetus for the development of gene t herapy strategies. A recombinant dystrophin cDNA and animal models of the disease are available for this therapy development. Characterizati on of these reagents and current progress toward gene therapy for DMD will be described.