title: Immune rejection of human dystrophin following intramuscular injections of naked DNA in mdx mice.
abstract: Intramuscular administration of plasmid expressing full-length human dystrophin in dystrophin-deficient adult mdx mice resulted in humoral and weak specific T cell responses against the human dystrophin protein. Following plasmid injection, human dystrophin was detected in the injected muscles at 7 days, but decreased thereafter. Anti-dystrophin antibodies were found 21 days following plasmid injection, which coincided with transient myositis. This immune rejection prevented the mice from expressing human dystrophin after a second plasmid injection. No anti-DNA antibodies were found. Anti-dystrophin antibodies were seen in a smaller proportion of plasmid-injected dystrophin-competent C57BL/10 mice, suggesting that the immune rejection of dystrophin may be explained partially by species differences in the dystrophin protein.
authors.affiliation: Transgène S.A., Strasbourg, France.
authors.forename: S
authors.initials: S
authors.lastname: Braun
authors.forename: C
authors.initials: C
authors.lastname: Thioudellet
authors.forename: P
authors.initials: P
authors.lastname: Rodriguez
authors.forename: D
authors.initials: D
authors.lastname: Ali-Hadji
authors.forename: F
authors.initials: F
authors.lastname: Perraud
authors.forename: N
authors.initials: N
authors.lastname: Accart
authors.forename: J M
authors.initials: JM
authors.lastname: Balloul
authors.forename: C
authors.initials: C
authors.lastname: Halluard
authors.forename: B
authors.initials: B
authors.lastname: Acres
authors.forename: B
authors.initials: B
authors.lastname: Cavallini
authors.forename: A
authors.initials: A
authors.lastname: Pavirani
mesh_terms: D000818:Animals; D000906:Antibodies / Q000032:analysis*; D015153:Blotting, Western / Q000379:methods; D016189:Dystrophin / Q000032:analysis / Q000235:genetics* / Q000276:immunology; D004797:Enzyme-Linked Immunosorbent Assay / Q000379:methods; D015316:Genetic Therapy / Q000009:adverse effects* / Q000379:methods; D006801:Humans; D007150:Immunohistochemistry / Q000379:methods; D007273:Injections, Intramuscular; D051379:Mice; D008810:Mice, Inbred C57BL; D018101:Mice, Inbred mdx; D018482:Muscle, Skeletal / Q000737:chemistry / Q000276:immunology; D020388:Muscular Dystrophy, Duchenne / Q000276:immunology / Q000628:therapy*; D010957:Plasmids / Q000008:administration & dosage*; D013045:Species Specificity; D013601:T-Lymphocytes / Q000276:immunology*
chemical_list: D000906:Antibodies; D016189:Dystrophin