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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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Main Authors: | , , , , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
National Academy of Sciences
2012
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Assuntos: | |
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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