Exon 45 Skipping Through U1-snRNA Antisense Molecules Recovers the Dys-nNOS Pathway and Muscle Differentiation in Human DMD Myoblasts

Exon skipping has been demonstrated to be a successful strategy for the gene therapy of Duchenne muscular dystrophy (DMD): the rational being to convert severe Duchenne forms into milder Becker ones. Here, we show the selection of U1 snRNA-antisense constructs able to confer effective rescue of dyst...

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Detalhes bibliográficos
Main Authors: Cazzella, Valentina, Martone, Julie, Pinnarò, Chiara, Santini, Tiziana, Twayana, Shyam Sundar, Sthandier, Olga, D'Amico, Adele, Ricotti, Valeria, Bertini, Enrico, Muntoni, Francesco, Bozzoni, Irene
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2012
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3498801/
https://ncbi.nlm.nih.gov/pubmed/22968481
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.178
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