Article
Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice.
Human molecular genetics - 1 Aug 1995
Phelps S F, Hauser M A, Cole N M, Rafael J A, Hinkle R T, Faulkner J A, Chamberlain J S
Abstract excerpt
Duchenne and Becker muscular dystrophy are caused by defects in the dystrophin gene, and are candidates for treatment by gene therapy. We have shown previously that overexpression of a full-length dystrophin cDNA prevents the development of dystrophic symptoms in mdx mice. We show here that this functional correction can be achieved by expressing the full-length muscle isoform at a lower level than is present in...
Topics
- Animals
- DNA, Complementary
- Diaphragm
- Dystrophin
- Female
- Gene Expression
- Genetic Therapy
- Genetic Vectors
- Humans
- Male
- Mice
- Mice, Inbred C57BL
