In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy

In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy

Delivery of therapeutic transgenes with adeno-associated viral (AAV) vectors for treatment of myopathies has yielded encouraging results in animal models and early clinical studies. Although certain AAV serotypes efficiently target muscle fibers, transduction of the muscle stem cells, also known as...

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Bibliographic Details
Main Authors: Jennifer B. Kwon, Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, Charles A. Gersbach
Format: Article
Language:English
Published: Elsevier 2020-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
dmd
satellite
pax7
gene-editing
crispr
aav
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120302011

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http://www.sciencedirect.com/science/article/pii/S2329050120302011

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