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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2019
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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) |
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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 |
| work_keys_str_mv |
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