Article
Human dystrophin expression corrects the myopathic phenotype in transgenic mdx mice.
Human molecular genetics - 1 Apr 1992
Wells D J, Wells K E, Walsh F S, Davies K E, Goldspink G, Love D R, Chan-Thomas P, Dunckley M G, Piper T, Dickson G
Abstract excerpt
Duchenne and the less severe Becker form of muscular dystrophy (DMD,BMD) result from genetic deficiency in the level and/or activity of the protein dystrophin. The recent availability of cDNA based minigenes encoding recombinant dystrophin polypeptides has raised the possibility of somatic gene t...
Topics
- Animals
- Blotting, Southern
- Blotting, Western
- Cloning, Molecular
- Creatine Kinase
- DNA
- Dystrophin
- Genetic Therapy
- Humans
- Male
- Mice
- Mice, Mutant Strains
- Mice, Transgenic
- Muscles
- Muscular Dystrophy, Animal
- Phenotype
- Plasmids
- Polymerase Chain Reaction