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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Bibliographic Details

Published in:	Molecular Therapy: Methods & Clinical Development
Main Authors:	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:	Article
Language:	English
Published:	Elsevier 2022-09-01
Subjects:	Duchenne muscular dystrophy Becker muscular dystrophy dystrophin exon skipping U7snRNA gene therapy
Online Access:	http://www.sciencedirect.com/science/article/pii/S2329050122000973