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Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration

Plasmid-mediated gene therapy can restore dystrophin expression in skeletal muscle in the mdx mouse, a model of Duchenne muscular dystrophy. However, sufficient long-term expression and distribution of dystrophin remain a hurdle for translating this technology into a viable treatment for Duchenne mu...

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Detalhes bibliográficos
Main Authors: Bertoni, Carmen, Jarrahian, Sohail, Wheeler, Thurman M., Li, Yining, Olivares, Eric C., Calos, Michele P., Rando, Thomas A.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2006
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1326153/
https://ncbi.nlm.nih.gov/pubmed/16387861
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0504505102
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