Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

Gaucher disease is a lysosomal storage disorder caused by insufficient glucocerebrosidase expression. Here, the authors describe a CRISPR/Cas9-based gene-editing approach to re-express this enzyme in human blood stem cells and show that they can engraft in NSG mice and differentiate into functional...

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Autores principales:	Samantha G. Scharenberg, Edina Poletto, Katherine L. Lucot, Pasqualina Colella, Adam Sheikali, Thomas J. Montine, Matthew H. Porteus, Natalia Gomez-Ospina
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2020
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/224d60d712dd4dc594a6f383d84a1ecd
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spelling	oai:doaj.org-article:224d60d712dd4dc594a6f383d84a1ecd2021-12-02T18:18:53ZEngineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing10.1038/s41467-020-17148-x2041-1723https://doaj.org/article/224d60d712dd4dc594a6f383d84a1ecd2020-07-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-17148-xhttps://doaj.org/toc/2041-1723Gaucher disease is a lysosomal storage disorder caused by insufficient glucocerebrosidase expression. Here, the authors describe a CRISPR/Cas9-based gene-editing approach to re-express this enzyme in human blood stem cells and show that they can engraft in NSG mice and differentiate into functional macrophages.Samantha G. ScharenbergEdina PolettoKatherine L. LucotPasqualina ColellaAdam SheikaliThomas J. MontineMatthew H. PorteusNatalia Gomez-OspinaNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-14 (2020)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Samantha G. Scharenberg Edina Poletto Katherine L. Lucot Pasqualina Colella Adam Sheikali Thomas J. Montine Matthew H. Porteus Natalia Gomez-Ospina Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
description	Gaucher disease is a lysosomal storage disorder caused by insufficient glucocerebrosidase expression. Here, the authors describe a CRISPR/Cas9-based gene-editing approach to re-express this enzyme in human blood stem cells and show that they can engraft in NSG mice and differentiate into functional macrophages.
format	article
author	Samantha G. Scharenberg Edina Poletto Katherine L. Lucot Pasqualina Colella Adam Sheikali Thomas J. Montine Matthew H. Porteus Natalia Gomez-Ospina
author_facet	Samantha G. Scharenberg Edina Poletto Katherine L. Lucot Pasqualina Colella Adam Sheikali Thomas J. Montine Matthew H. Porteus Natalia Gomez-Ospina
author_sort	Samantha G. Scharenberg
title	Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
title_short	Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
title_full	Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
title_fullStr	Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
title_full_unstemmed	Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
title_sort	engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
publisher	Nature Portfolio
publishDate	2020
url	https://doaj.org/article/224d60d712dd4dc594a6f383d84a1ecd
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Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing

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