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
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/66cf921ac2ec4178bebe47ca77ff7c87
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spelling	oai:doaj.org-article:66cf921ac2ec4178bebe47ca77ff7c872021-12-02T16:57:21ZGene correction for SCID-X1 in long-term hematopoietic stem cells10.1038/s41467-019-09614-y2041-1723https://doaj.org/article/66cf921ac2ec4178bebe47ca77ff7c872019-04-01T00:00:00Zhttps://doi.org/10.1038/s41467-019-09614-yhttps://doaj.org/toc/2041-1723Gene 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.Mara Pavel-DinuVolker WiebkingBeruh T. DejeneWaracharee SrifaSruthi MantriCarmencita E. NicolasCiaran LeeGang BaoEric J. KildebeckNiraj PunjyaCamille SindhuMatthew A. InlayNivedita SaxenaSuk See DeRavinHarry MalechMaria Grazia RoncaroloKenneth I. WeinbergMatthew H. PorteusNature PortfolioarticleScienceQENNature Communications, Vol 10, Iss 1, Pp 1-15 (2019)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q 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 Gene correction for SCID-X1 in long-term hematopoietic stem cells
description	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.
format	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
author_facet	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
author_sort	Mara Pavel-Dinu
title	Gene correction for SCID-X1 in long-term hematopoietic stem cells
title_short	Gene correction for SCID-X1 in long-term hematopoietic stem cells
title_full	Gene correction for SCID-X1 in long-term hematopoietic stem cells
title_fullStr	Gene correction for SCID-X1 in long-term hematopoietic stem cells
title_full_unstemmed	Gene correction for SCID-X1 in long-term hematopoietic stem cells
title_sort	gene correction for scid-x1 in long-term hematopoietic stem cells
publisher	Nature Portfolio
publishDate	2019
url	https://doaj.org/article/66cf921ac2ec4178bebe47ca77ff7c87
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Gene correction for SCID-X1 in long-term hematopoietic stem cells

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