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Hum Gene Ther ; 15(8): 770-82, 2004 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-15319034

RESUMO

Our previous studies have demonstrated that the intraarterial delivery of naked plasmid DNA leads to high levels of foreign gene expression throughout the muscles of the targeted limb. Although the procedure was first developed in rats and then extended to nonhuman primates, the present study has successfully implemented the procedure in normal mice and the mdx mouse model for Duchenne muscular dystrophy. After intraarterial delivery of plasmid DNA expressing the normal, full-length mouse dystrophin from either the cytomegalovirus promoter or a muscle-specific human desmin gene control region, mdx mouse muscle stably expressed dystrophin in 1-5% of the myofibers of the injected hind limb for at least 6 months. This expression generated an antibody response but no apparent cellular response.


Assuntos
DNA/genética , Distrofina/metabolismo , Expressão Gênica , Terapia Genética/métodos , Distrofia Muscular de Duchenne/terapia , Animais , Creatina Quinase/sangue , Distrofina/genética , Distrofina/imunologia , Eletroforese em Gel de Poliacrilamida , Immunoblotting , Imuno-Histoquímica , Injeções Intra-Arteriais , Camundongos , Camundongos Endogâmicos mdx , Músculo Esquelético/metabolismo , Plasmídeos/genética
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