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Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice

Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease caused by an absence of the dystrophin protein, which is essential for muscle fiber integrity. Among the developed therapeutic strategies for DMD, the exon-skipping approach corrects the frameshift and partially restores dystro...

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Detalles Bibliográficos
Autores principales:	Forand, Anne, Muchir, Antoine, Mougenot, Nathalie, Sevoz-Couche, Caroline, Peccate, Cécile, Lemaitre, Mégane, Izabelle, Charlotte, Wood, Matthew, Lorain, Stéphanie, Piétri-Rouxel, France
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/PMC7177166/ https://www.ncbi.nlm.nih.gov/pubmed/32346547 http://dx.doi.org/10.1016/j.omtm.2020.03.011

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