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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Principais autores:	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
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	Elsevier 2022-09-01
סדרה:	Molecular Therapy: Methods & Clinical Development
נושאים:	Duchenne muscular dystrophy Becker muscular dystrophy dystrophin exon skipping U7snRNA gene therapy
גישה מקוונת:	http://www.sciencedirect.com/science/article/pii/S2329050122000973
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