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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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Autores principales: Fletcher, Sue, Adams, Abbie M, Johnsen, Russell D, Greer, Kane, Moulton, Hong M, Wilton, Steve D
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2010
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Acceso en línea: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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