Exon Skipping and Duchenne Muscular Dystrophy Therapy: Selection of the Most Active U1 snRNA Antisense Able to Induce Dystrophin Exon 51 Skipping

One promising approach for the gene therapy of Duchenne muscular dystrophy (DMD) is exon skipping. When thinking of possible intervention on human, it is very crucial to identify the most appropriate antisense sequences able to provide the highest possible skipping efficiency. In this article, we co...

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Detalhes bibliográficos
Main Authors: Incitti, Tania, De Angelis, Fernanda G, Cazzella, Valentina, Sthandier, Olga, Pinnarò, Chiara, Legnini, Ivano, Bozzoni, Irene
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2010
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2956936/
https://ncbi.nlm.nih.gov/pubmed/20551908
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.123
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