title: Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene.
abstract: 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.
authors.affiliation: Unité 129 INSERM, Institut Cochin de Génétique Moléculaire (ICGM), Paris, France.
authors.forename: N
authors.initials: N
authors.lastname: Vincent
authors.forename: T
authors.initials: T
authors.lastname: Ragot
authors.forename: H
authors.initials: H
authors.lastname: Gilgenkrantz
authors.forename: D
authors.initials: D
authors.lastname: Couton
authors.forename: P
authors.initials: P
authors.lastname: Chafey
authors.forename: A
authors.initials: A
authors.lastname: Grégoire
authors.forename: P
authors.initials: P
authors.lastname: Briand
authors.forename: J C
authors.initials: JC
authors.lastname: Kaplan
authors.forename: A
authors.initials: A
authors.lastname: Kahn
authors.forename: M
authors.initials: M
authors.lastname: Perricaudet
mesh_terms: 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
chemical_list: D016189:Dystrophin; D001616:beta-Galactosidase