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 a...
Topics
- Animals
- Cloning, Molecular
- Dystrophin
- Forecasting
- Genetic Therapy
- Humans
- Muscular Dystrophies
- Mutation
- Sex Chromosome Aberrations
- X Chromosome