Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing

Antisense oligomer-induced manipulation of dystrophin pre-mRNA processing can remove exons carrying mutations, or exclude exons flanking frameshifting mutations, and restore dystrophin expression in dystrophinopathy models and in Duchenne muscular dystrophy (DMD) patients. Splice intervention can al...

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Autori principali: Fletcher, Sue, Adams, Abbie M, Johnsen, Russell D, Greer, Kane, Moulton, Hong M, Wilton, Steve D
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2010
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2889736/
https://ncbi.nlm.nih.gov/pubmed/20332768
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.45
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