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

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

Gene correction in hematopoietic stem cells could be a powerful way to treat monogenic diseases of the blood and immune system. Here the authors develop a strategy using CRISPR-Cas9 and an aAdeno-Associated vVirus(AAV)-delivered IL2RG cDNA to correct X-linked sSevere Ccombined iImmunodeficiency (SCI...

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Autores principales: Mara Pavel-Dinu, Volker Wiebking, Beruh T. Dejene, Waracharee Srifa, Sruthi Mantri, Carmencita E. Nicolas, Ciaran Lee, Gang Bao, Eric J. Kildebeck, Niraj Punjya, Camille Sindhu, Matthew A. Inlay, Nivedita Saxena, Suk See DeRavin, Harry Malech, Maria Grazia Roncarolo, Kenneth I. Weinberg, Matthew H. Porteus
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2019
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Science
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Acceso en línea:https://doaj.org/article/66cf921ac2ec4178bebe47ca77ff7c87
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