Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity

Imperfect use of dystrophin causes Duchenne and Becker muscular dystrophy, which are X-linked diseases, and gene therapy may be adopted by using a full-length dystrophin transgene to correct dystrophic symptoms in mdx mice. Dystrophic symptoms from mdx muscles could be removed by the high levels of dystrophin which transgenic mice create. In the case of transgenic mice, muscular dystrophy could be corrected by using tissue specific full-length dystrophics cDNA clone. The problems in the mice were almost corrected but the total cure was not possible either due to low protein use or defects in the truncated dystrophin clone.