Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

CRISPR mediated gene correction of sickle cell disease (SCD) in patient-derived hematopoietic stem cells is a promising avenue for therapy. Here the authors use a humanized SCD mouse model to study gene editing in the context of autologous transplantation.

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Autores principales:	Adam C. Wilkinson, Daniel P. Dever, Ron Baik, Joab Camarena, Ian Hsu, Carsten T. Charlesworth, Chika Morita, Hiromitsu Nakauchi, Matthew H. Porteus
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/33cf7e3f25534b81a1ff9bc124c467e5
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spelling	oai:doaj.org-article:33cf7e3f25534b81a1ff9bc124c467e52021-12-02T14:16:14ZCas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice10.1038/s41467-021-20909-x2041-1723https://doaj.org/article/33cf7e3f25534b81a1ff9bc124c467e52021-01-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-20909-xhttps://doaj.org/toc/2041-1723CRISPR mediated gene correction of sickle cell disease (SCD) in patient-derived hematopoietic stem cells is a promising avenue for therapy. Here the authors use a humanized SCD mouse model to study gene editing in the context of autologous transplantation.Adam C. WilkinsonDaniel P. DeverRon BaikJoab CamarenaIan HsuCarsten T. CharlesworthChika MoritaHiromitsu NakauchiMatthew H. PorteusNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-9 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Adam C. Wilkinson Daniel P. Dever Ron Baik Joab Camarena Ian Hsu Carsten T. Charlesworth Chika Morita Hiromitsu Nakauchi Matthew H. Porteus Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
description	CRISPR mediated gene correction of sickle cell disease (SCD) in patient-derived hematopoietic stem cells is a promising avenue for therapy. Here the authors use a humanized SCD mouse model to study gene editing in the context of autologous transplantation.
format	article
author	Adam C. Wilkinson Daniel P. Dever Ron Baik Joab Camarena Ian Hsu Carsten T. Charlesworth Chika Morita Hiromitsu Nakauchi Matthew H. Porteus
author_facet	Adam C. Wilkinson Daniel P. Dever Ron Baik Joab Camarena Ian Hsu Carsten T. Charlesworth Chika Morita Hiromitsu Nakauchi Matthew H. Porteus
author_sort	Adam C. Wilkinson
title	Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
title_short	Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
title_full	Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
title_fullStr	Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
title_full_unstemmed	Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
title_sort	cas9-aav6 gene correction of beta-globin in autologous hscs improves sickle cell disease erythropoiesis in mice
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/33cf7e3f25534b81a1ff9bc124c467e5
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Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

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