학술논문
Gene correction for SCID-X1 in long-term hematopoietic stem cells
Document Type
article
Author
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
Source
Nature Communications, Vol 10, Iss 1, Pp 1-15 (2019)
Subject
Language
English
ISSN
2041-1723
Abstract
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 (SCID-X1) with a high success rate.