Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping

Duchenne muscular dystrophy (DMD), one of the most severe neuromuscular disorders of childhood, is caused by the absence of a functional dystrophin. Antisense oligomer (AO) induced exon skipping is being investigated to restore functional dystrophin expression in models of muscular dystrophy and DMD...

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Bibliographic Details
Main Authors: Mitrpant, Chalermchai, Adams, Abbie M, Meloni, Penny L, Muntoni, Francesco, Fletcher, Sue, Wilton, Steve D
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2009
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Original Articles
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835229/
https://ncbi.nlm.nih.gov/pubmed/19293776
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.49
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