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 musc le disorder, caused by mutations in the dystrophin gene. We have inves tigated adenovirus-mediated transfer of a dystrophin minigene in a mut ant mouse lacking dystrophin, the mdx mouse. We report here that six m onths after a single intramuscular injection of a recombinant adenovir us containing a human dystrophin minigene, a large number of dystrophi n-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 proce ss that affects the dystrophin-deficient mdx myofibres.