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Base editing repairs an SGCA mutation in human primary muscle stem cells

Skeletal muscle can regenerate from muscle stem cells and their myogenic precursor cell progeny, myoblasts. However, precise gene editing in human muscle stem cells for autologous cell replacement therapies of untreatable genetic muscle diseases has not yet been reported. Loss-of-function mutations...

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Detalles Bibliográficos
Autores principales:	Escobar, Helena, Krause, Anne, Keiper, Sandra, Kieshauer, Janine, Müthel, Stefanie, de Paredes, Manuel García, Metzler, Eric, Kühn, Ralf, Heyd, Florian, Spuler, Simone
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Clinical Investigation 2021
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8262330/ https://www.ncbi.nlm.nih.gov/pubmed/33848270 http://dx.doi.org/10.1172/jci.insight.145994

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