Nature Communications (Apr 2019)
Gene correction for SCID-X1 in long-term hematopoietic stem cells
- 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
Affiliations
- Mara Pavel-Dinu
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Volker Wiebking
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Beruh T. Dejene
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Waracharee Srifa
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Sruthi Mantri
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Carmencita E. Nicolas
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Ciaran Lee
- Department of Bioengineering, Rice University
- Gang Bao
- Department of Bioengineering, Rice University
- Eric J. Kildebeck
- Center for Engineering Innovation, University of Texas at Dallas
- Niraj Punjya
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Camille Sindhu
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Matthew A. Inlay
- Department of Cellular and Molecular Biosciences, University of California Irvine
- Nivedita Saxena
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Suk See DeRavin
- Laboratory of Host Defenses, National Institutes of Allergy and Infectious Diseases, National Institute of Health
- Harry Malech
- Laboratory of Host Defenses, National Institutes of Allergy and Infectious Diseases, National Institute of Health
- Maria Grazia Roncarolo
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Kenneth I. Weinberg
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- Matthew H. Porteus
- Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University
- DOI
- https://doi.org/10.1038/s41467-019-09614-y
- Journal volume & issue
-
Vol. 10,
no. 1
pp. 1 – 15
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.
