Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

OBJECTIVE: Duchenne muscular dystrophy (DMD) is caused by the inability to produce dystrophin protein at the myofiber membrane. A method to rescue dystrophin production by antisense oligonucleotides, termed `exon-skipping', has been reported for the mdx mouse and in four DMD patients by local i...

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Udgivet i:Ann Neurol
Main Authors: Yokota, Toshifumi, Lu, Qi-long, Partridge, Terence, Kobayashi, Masanori, Nakamura, Akinori, Takeda, Shin'ichi, Hoffman, Eric
Format: Artigo
Sprog:Inglês
Udgivet: 2009
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5951302/
https://ncbi.nlm.nih.gov/pubmed/19288467
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.21627
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