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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Detaylı Bibliyografya
Asıl Yazarlar: Philippine Aupy, Faouzi Zarrouki, Quentin Sandro, Cécile Gastaldi, Pierre-Olivier Buclez, Kamel Mamchaoui, Luis Garcia, Cyrille Vaillend, Aurélie Goyenvalle
Materyal Türü: Makale
Dil:English
Baskı/Yayın Bilgisi: Elsevier 2020-06-01
Seri Bilgileri:Molecular Therapy: Methods & Clinical Development
Konular:
exon skipping
U7snRNA
adeno-associated viral vector
gene therapy
Duchenne muscular dystrophy
mouse model
Online Erişim:http://www.sciencedirect.com/science/article/pii/S2329050120300851

Internet

http://www.sciencedirect.com/science/article/pii/S2329050120300851

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