Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

Duchenne muscular dystrophy (DMD) is typically caused by mutations that disrupt the DMD reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an internal ribosomal entry site (IRES) in exon 5, driving expression of a highly functional N-truncated dystrophin. We have...

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Autors principals:	Nicolas Wein, Tatyana A. Vetter, Adeline Vulin, Tabatha R. Simmons, Emma C. Frair, Adrienne J. Bradley, Liubov V. Gushchina, Camila F. Almeida, Nianyuan Huang, Daniel Lesman, Dhanarajan Rajakumar, Robert B. Weiss, Kevin M. Flanigan
Format:	Artigo
Idioma:	Inglês
Publicat:	Elsevier 2022-09-01
Col·lecció:	Molecular Therapy: Methods & Clinical Development
Matèries:	Duchenne muscular dystrophy Becker muscular dystrophy dystrophin exon skipping U7snRNA gene therapy
Accés en línia:	http://www.sciencedirect.com/science/article/pii/S2329050122000973
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