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Gene correction for SCID-X1 in long-term hematopoietic stem cells

Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. Here we describe an approach for X-linked sSevere cCombined iImmunodeficiency (SCID-X1) using targeted integration of a cDNA into the endogenous...

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Detalles Bibliográficos
Autores principales:	Pavel-Dinu, Mara, Wiebking, Volker, Dejene, Beruh T., Srifa, Waracharee, Mantri, Sruthi, Nicolas, Carmencita E., Lee, Ciaran, Bao, Gang, Kildebeck, Eric J., Punjya, Niraj, Sindhu, Camille, Inlay, Matthew A., Saxena, Nivedita, DeRavin, Suk See, Malech, Harry, Roncarolo, Maria Grazia, Weinberg, Kenneth I., Porteus, Matthew H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6456568/ https://www.ncbi.nlm.nih.gov/pubmed/30967552 http://dx.doi.org/10.1038/s41467-019-09614-y

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