Article
Gene therapy prospects for Duchenne muscular dystrophy.
European neurology - 1 Jan 1994
Clemens P R, Caskey C T
Abstract excerpt
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...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.