Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

In recent years, hematopoietic stem cells gene editing has emerged as a promising tool to treat blood disorders. Here the authors develop a CRISPR/Cas9-based genome editing strategy that allows the precise correction of Wiskott-Aldrich Syndrome in vitro and in vivo with high efficiency.

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Autores principales:	Rajeev Rai, Marianna Romito, Elizabeth Rivers, Giandomenico Turchiano, Georges Blattner, Winston Vetharoy, Dariusz Ladon, Geoffroy Andrieux, Fang Zhang, Marta Zinicola, Diego Leon-Rico, Giorgia Santilli, Adrian J. Thrasher, Alessia Cavazza
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2020
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/e93094c9c51f4c1aa746e611fd6ea64c
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spelling	oai:doaj.org-article:e93094c9c51f4c1aa746e611fd6ea64c2021-12-02T16:27:51ZTargeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome10.1038/s41467-020-17626-22041-1723https://doaj.org/article/e93094c9c51f4c1aa746e611fd6ea64c2020-08-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-17626-2https://doaj.org/toc/2041-1723In recent years, hematopoietic stem cells gene editing has emerged as a promising tool to treat blood disorders. Here the authors develop a CRISPR/Cas9-based genome editing strategy that allows the precise correction of Wiskott-Aldrich Syndrome in vitro and in vivo with high efficiency.Rajeev RaiMarianna RomitoElizabeth RiversGiandomenico TurchianoGeorges BlattnerWinston VetharoyDariusz LadonGeoffroy AndrieuxFang ZhangMarta ZinicolaDiego Leon-RicoGiorgia SantilliAdrian J. ThrasherAlessia CavazzaNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-15 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Rajeev Rai Marianna Romito Elizabeth Rivers Giandomenico Turchiano Georges Blattner Winston Vetharoy Dariusz Ladon Geoffroy Andrieux Fang Zhang Marta Zinicola Diego Leon-Rico Giorgia Santilli Adrian J. Thrasher Alessia Cavazza Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
description	In recent years, hematopoietic stem cells gene editing has emerged as a promising tool to treat blood disorders. Here the authors develop a CRISPR/Cas9-based genome editing strategy that allows the precise correction of Wiskott-Aldrich Syndrome in vitro and in vivo with high efficiency.
format	article
author	Rajeev Rai Marianna Romito Elizabeth Rivers Giandomenico Turchiano Georges Blattner Winston Vetharoy Dariusz Ladon Geoffroy Andrieux Fang Zhang Marta Zinicola Diego Leon-Rico Giorgia Santilli Adrian J. Thrasher Alessia Cavazza
author_facet	Rajeev Rai Marianna Romito Elizabeth Rivers Giandomenico Turchiano Georges Blattner Winston Vetharoy Dariusz Ladon Geoffroy Andrieux Fang Zhang Marta Zinicola Diego Leon-Rico Giorgia Santilli Adrian J. Thrasher Alessia Cavazza
author_sort	Rajeev Rai
title	Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
title_short	Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
title_full	Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
title_fullStr	Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
title_full_unstemmed	Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
title_sort	targeted gene correction of human hematopoietic stem cells for the treatment of wiskott - aldrich syndrome
publisher	Nature Portfolio
publishDate	2020
url	https://doaj.org/article/e93094c9c51f4c1aa746e611fd6ea64c
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Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

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