[Therapeutic strategy for muscular dystrophies].
A successful cure for muscular dystrophies has yet to be demonstrated, but we could notice spectacular progress for the past few years. Advances have been made in methods of delivering foreign genes into muscle cells, either as naked plasmid DNA or via viral vectors. Among the virus-based methods, results with adeno-associated viral vectors have been the most promising. Systemic administration of anti-sense oligonucleotides has shown incredible success in inducing dystrophin expression and ameliorating muscle pathology via exon skipping in canine models of muscular dystrophy. Biochemical and functional rescue of dystrophic muscle has also been demonstrated with the transplantation of normal or genetically modified myogenic stem cells, as well as with a number of pharmacological approaches in animal models. Here we review the current researches on the treatment of muscular dystrophies and clinical trials on Duchenne muscular dystrophy.
Shin
Jin-Hong
JH
Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi-machi, Kodaira, Tokyo, Japan.
Takeda
Shin'ichi
S
D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000379:methods; D004285:Dogs; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D009136:Muscular Dystrophies / Q000188:drug therapy / Q000628:therapy*