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Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery

Duchenne muscular dystrophy (DMD), the commonest form of muscular dystrophy, is caused by lack of dystrophin. One of the most promising therapeutic approaches is antisense-mediated elimination of frame-disrupting mutations by exon skipping. However, this approach faces two major hurdles: limited app...

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Detalhes bibliográficos
Main Authors: Aoki, Yoshitsugu, Yokota, Toshifumi, Nagata, Tetsuya, Nakamura, Akinori, Tanihata, Jun, Saito, Takashi, Duguez, Stephanie M. R., Nagaraju, Kanneboyina, Hoffman, Eric P., Partridge, Terence, Takeda, Shin'ichi
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2012
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3427064/
https://ncbi.nlm.nih.gov/pubmed/22869723
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1204638109
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