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The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is associated with mutations in the dystrophin gene that disrupt the open reading frame whereas the milder Becker's form is associated with mutations which leave an in-frame mRNA transcript that can be translated into a protein that includes the N- and C- termi...

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Bibliografiske detaljer
Main Authors: Lu, Qi-Long, Yokota, Toshifumi, Takeda, Shin'ichi, Garcia, Luis, Muntoni, Francesco, Partridge, Terence
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2011
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3017449/
https://ncbi.nlm.nih.gov/pubmed/20978473
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.219
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