Long–term correction of mouse dystrophic degeneration by adenovirus–mediated transfer of a minidystrophin gene

Duchenne muscular dystrophy (DMD) is a fatal progressive X–linked muscle disorder, caused by mutations in the dystrophin gene. We have investigated adenovirus–mediated transfer of a dystrophin minigene in a mutant mouse lacking dystrophin, the mdx mouse. We report here that six months after a single intramuscular injection of a recombinant adenovirus containing a human dystrophin minigene, a large number of dystrophin–positive fibres are still detected in the injected muscles. Moreover, although the minigene encodes a truncated protein, its expression is able to protect the fibres efficiently against the degeneration process that affects the dystrophin–deficient mdx myofibres.