Gene therapy prospects for Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disorder caused by mutations in the dystrophin gene. The lack of adequate therapy for this disease provides impetus for the development of gene therapy strategies. A recombinant dystrophin cDNA and animal models of the disease are available for this therapy development. Characterization of these reagents and current progress toward gene therapy for DMD will be described.
Clemens
P R
PR
Institute for Molecular Genetics, Baylor College of Medicine, Houston, TX 77030.
Caskey
C T
CT
D000818:Animals; D003001:Cloning, Molecular; D016189:Dystrophin / Q000235:genetics; D005544:Forecasting; D015316:Genetic Therapy; D006801:Humans; D009136:Muscular Dystrophies / Q000235:genetics / Q000628:therapy*; D009154:Mutation; D012729:Sex Chromosome Aberrations / Q000235:genetics; D014960:X Chromosome
D016189:Dystrophin