AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy

In the context of future adeno-associated viral (AAV)-based clinical trials for Duchenne myopathy, AAV genome fate in dystrophic muscles is of importance considering the viral capsid immunogenicity that prohibits recurring treatments. We showed that AAV genomes encoding non-therapeutic U7 were lost...

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Detalles Bibliográficos
Autores principales:	Le Hir, M, Goyenvalle, A, Peccate, C, Précigout, G, Davies, K, Voit, T, Garcia, L, Lorain, S
Formato:	Journal article
Lenguaje:	English
Publicado:	2013

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