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Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

Gene therapy and antisense approaches hold promise for the treatment of Duchenne muscular dystrophy (DMD). The advantages of both therapeutic strategies can be combined by vectorizing antisense sequences into an adeno-associated virus (AAV) vector. We previously reported the efficacy of AAV-U7 small...

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Detalles Bibliográficos
Autores principales:	Aupy, Philippine, Zarrouki, Faouzi, Sandro, Quentin, Gastaldi, Cécile, Buclez, Pierre-Olivier, Mamchaoui, Kamel, Garcia, Luis, Vaillend, Cyrille, Goyenvalle, Aurélie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7240049/ https://www.ncbi.nlm.nih.gov/pubmed/32462052 http://dx.doi.org/10.1016/j.omtm.2020.04.025

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