Duchenne muscular dystrophy: current knowledge, treatment, and future prospects.
The cloning of the dystrophin gene has led to major advances in the understanding of the molecular genetic basis of Duchenne, Becker, and other muscular dystrophies associated with mutations in genes encoding members of the dystrophin-associated glycoprotein complex. The recent introduction of pharmaceutical agents such as prednisone has shown great promise in delaying the progression of Duchenne muscular dystrophy but there remains a need to develop more long-term therapeutic interventions. Knowledge of the nature of the dystrophin gene and the glycoprotein complex has led many researchers to think that somatic gene replacement represents the most promising approach to treatment. The potential use of this strategy has been shown in the mdx mouse model of Duchenne muscular dystrophy, where germ line gene transfer of either a full-length or a smaller Becker-type dystrophin minigene prevents necrosis and restores normal muscle function.
Biggar
W Douglas
WD
Bloorview MacMillan Children's Centre and Department of Paediatrics, University of Toronto, Ontario, Canada.
Klamut
Henry J
HJ
Demacio
Paula C
PC
Stevens
Daniel J
DJ
Ray
Peter N
PN
D000818:Animals; D017690:Cell Transplantation; D003598:Cytoskeletal Proteins / Q000502:physiology; D016189:Dystrophin / Q000235:genetics / Q000502:physiology; D015316:Genetic Therapy; D006801:Humans; D008297:Male; D018482:Muscle, Skeletal / Q000166:cytology / Q000503:physiopathology / Q000637:transplantation; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis / Q000235:genetics / Q000503:physiopathology / Q000628:therapy
D003598:Cytoskeletal Proteins; D016189:Dystrophin