Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene.
Duchene 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.
Vincent
N
N
Unité 129 INSERM, Institut Cochin de Génétique Moléculaire (ICGM), Paris, France.
Ragot
T
T
Gilgenkrantz
H
H
Couton
D
D
Chafey
P
P
Grégoire
A
A
Briand
P
P
Kaplan
J C
JC
Kahn
A
A
Perricaudet
M
M
D000256:Adenoviridae / Q000235:genetics*; D000818:Animals; D016189:Dystrophin / Q000235:genetics*; D005814:Genes, Viral; D015316:Genetic Therapy; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic; D009136:Muscular Dystrophies / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D013997:Time Factors; D014162:Transfection; D001616:beta-Galactosidase / Q000235:genetics
D016189:Dystrophin; D001616:beta-Galactosidase