Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

Mucopolysaccharidosis type I (MPSI) is a lysosomal storage disease caused by insufficient iduronidase (IDUA) activity. Here, the authors use an ex vivo genome editing approach to overexpress IDUA in human hematopoietic stem and progenitor cells and show it can phenotypically correct MSPI in mouse mo...

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Autores principales: Natalia Gomez-Ospina, Samantha G. Scharenberg, Nathalie Mostrel, Rasmus O. Bak, Sruthi Mantri, Rolen M. Quadros, Channabasavaiah B. Gurumurthy, Ciaran Lee, Gang Bao, Carlos J. Suarez, Shaukat Khan, Kazuki Sawamoto, Shunji Tomatsu, Nitin Raj, Laura D. Attardi, Laure Aurelian, Matthew H. Porteus
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Publicado: Nature Portfolio 2019
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Acceso en línea:https://doaj.org/article/8f066f14a98c44ea90d2d9109e701772
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spelling oai:doaj.org-article:8f066f14a98c44ea90d2d9109e7017722021-12-02T16:58:04ZHuman genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I10.1038/s41467-019-11962-82041-1723https://doaj.org/article/8f066f14a98c44ea90d2d9109e7017722019-09-01T00:00:00Zhttps://doi.org/10.1038/s41467-019-11962-8https://doaj.org/toc/2041-1723Mucopolysaccharidosis type I (MPSI) is a lysosomal storage disease caused by insufficient iduronidase (IDUA) activity. Here, the authors use an ex vivo genome editing approach to overexpress IDUA in human hematopoietic stem and progenitor cells and show it can phenotypically correct MSPI in mouse model.Natalia Gomez-OspinaSamantha G. ScharenbergNathalie MostrelRasmus O. BakSruthi MantriRolen M. QuadrosChannabasavaiah B. GurumurthyCiaran LeeGang BaoCarlos J. SuarezShaukat KhanKazuki SawamotoShunji TomatsuNitin RajLaura D. AttardiLaure AurelianMatthew H. PorteusNature PortfolioarticleScienceQENNature Communications, Vol 10, Iss 1, Pp 1-14 (2019)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Natalia Gomez-Ospina
Samantha G. Scharenberg
Nathalie Mostrel
Rasmus O. Bak
Sruthi Mantri
Rolen M. Quadros
Channabasavaiah B. Gurumurthy
Ciaran Lee
Gang Bao
Carlos J. Suarez
Shaukat Khan
Kazuki Sawamoto
Shunji Tomatsu
Nitin Raj
Laura D. Attardi
Laure Aurelian
Matthew H. Porteus
Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
description Mucopolysaccharidosis type I (MPSI) is a lysosomal storage disease caused by insufficient iduronidase (IDUA) activity. Here, the authors use an ex vivo genome editing approach to overexpress IDUA in human hematopoietic stem and progenitor cells and show it can phenotypically correct MSPI in mouse model.
format article
author Natalia Gomez-Ospina
Samantha G. Scharenberg
Nathalie Mostrel
Rasmus O. Bak
Sruthi Mantri
Rolen M. Quadros
Channabasavaiah B. Gurumurthy
Ciaran Lee
Gang Bao
Carlos J. Suarez
Shaukat Khan
Kazuki Sawamoto
Shunji Tomatsu
Nitin Raj
Laura D. Attardi
Laure Aurelian
Matthew H. Porteus
author_facet Natalia Gomez-Ospina
Samantha G. Scharenberg
Nathalie Mostrel
Rasmus O. Bak
Sruthi Mantri
Rolen M. Quadros
Channabasavaiah B. Gurumurthy
Ciaran Lee
Gang Bao
Carlos J. Suarez
Shaukat Khan
Kazuki Sawamoto
Shunji Tomatsu
Nitin Raj
Laura D. Attardi
Laure Aurelian
Matthew H. Porteus
author_sort Natalia Gomez-Ospina
title Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
title_short Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
title_full Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
title_fullStr Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
title_full_unstemmed Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
title_sort human genome-edited hematopoietic stem cells phenotypically correct mucopolysaccharidosis type i
publisher Nature Portfolio
publishDate 2019
url https://doaj.org/article/8f066f14a98c44ea90d2d9109e701772
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