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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Detalles Bibliográficos
Autores principales: Kwon, Jennifer B., Ettyreddy, Adarsh R., Vankara, Ashish, Bohning, Joel D., Devlin, Garth, Hauschka, Stephen D., Asokan, Aravind, Gersbach, Charles A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7581966/
https://www.ncbi.nlm.nih.gov/pubmed/33145368
http://dx.doi.org/10.1016/j.omtm.2020.09.016

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