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Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping

Exon skipping mediated by tricyclo-DNA (tc-DNA) antisense oligonucleotides has been shown to induce significant levels of dystrophin restoration in mdx, a mouse model of Duchenne muscular dystrophy. This translates into significant improvement in key disease indicators in muscle, cardio-respiratory...

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Detalles Bibliográficos
Autores principales:	Omairi, Saleh, Hau, Kwan-Leong, Collin-Hooper, Henry, Montanaro, Federica, Goyenvalle, Aurelie, Garcia, Luis, Patel, Ketan
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6114118/ https://www.ncbi.nlm.nih.gov/pubmed/29246319 http://dx.doi.org/10.1016/j.omtn.2017.10.014

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