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...

ver descrição completa

Detalhes bibliográficos

Principais autores:	Jennifer B. Kwon, Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, Charles A. Gersbach
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2020-12-01
coleção:	Molecular Therapy: Methods & Clinical Development
Assuntos:	dmd satellite pax7 gene-editing crispr aav
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2329050120302011
Tags:	Adicionar Tag Sem tags, seja o primeiro a adicionar uma tag!