Extensive and Prolonged Restoration of Dystrophin Expression with Vivo-Morpholino-Mediated Multiple Exon Skipping in Dystrophic Dogs

Duchenne muscular dystrophy (DMD) is a severe and the most prevalent form of muscular dystrophy, characterized by rapid progression of muscle degeneration. Antisense-mediated exon skipping is currently one of the most promising therapeutic options for DMD. However, unmodified antisense oligos such a...

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Bibliografiske detaljer
Main Authors: Yokota, Toshifumi, Nakamura, Akinori, Nagata, Tetsuya, Saito, Takashi, Kobayashi, Masanori, Aoki, Yoshitsugu, Echigoya, Yusuke, Partridge, Terence, Hoffman, Eric P., Takeda, Shin'ichi
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2012
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Original Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3464409/
https://ncbi.nlm.nih.gov/pubmed/22888777
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2012.0368
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