Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

Gene therapy and antisense approaches hold promise for the treatment of Duchenne muscular dystrophy (DMD). The advantages of both therapeutic strategies can be combined by vectorizing antisense sequences into an adeno-associated virus (AAV) vector. We previously reported the efficacy of AAV-U7 small...

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Bibliografische gegevens
Hoofdauteurs:	Philippine Aupy, Faouzi Zarrouki, Quentin Sandro, Cécile Gastaldi, Pierre-Olivier Buclez, Kamel Mamchaoui, Luis Garcia, Cyrille Vaillend, Aurélie Goyenvalle
Formaat:	Artikel
Taal:	English
Gepubliceerd in:	Elsevier 2020-06-01
Reeks:	Molecular Therapy: Methods & Clinical Development
Onderwerpen:	exon skipping U7snRNA adeno-associated viral vector gene therapy Duchenne muscular dystrophy mouse model
Online toegang:	http://www.sciencedirect.com/science/article/pii/S2329050120300851

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