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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Bibliografiske detaljer
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
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2012
Fag:
Biological Sciences
Online adgang: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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