Nature Communications (Sep 2019)
Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
- 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
Affiliations
- Natalia Gomez-Ospina
- Department of Pediatrics, Stanford University School of Medicine
- Samantha G. Scharenberg
- Department of Pediatrics, Stanford University School of Medicine
- Nathalie Mostrel
- Department of Pediatrics, Stanford University School of Medicine
- Rasmus O. Bak
- Department of Biomedicine, Aarhus University
- Sruthi Mantri
- Department of Pediatrics, Stanford University School of Medicine
- Rolen M. Quadros
- Mouse Genome Engineering Core Facility, Vice Chancellor for Research Office, University of Nebraska Medical Center
- Channabasavaiah B. Gurumurthy
- Mouse Genome Engineering Core Facility, Vice Chancellor for Research Office, University of Nebraska Medical Center
- Ciaran Lee
- Department of Bioengineering, Rice University
- Gang Bao
- Department of Bioengineering, Rice University
- Carlos J. Suarez
- Department of Pathology, Stanford University School of Medicine
- Shaukat Khan
- Nemours/ Alfred I. duPont Hospital for Children
- Kazuki Sawamoto
- Nemours/ Alfred I. duPont Hospital for Children
- Shunji Tomatsu
- Nemours/ Alfred I. duPont Hospital for Children
- Nitin Raj
- Department of Radiation Oncology, Stanford University School of Medicine
- Laura D. Attardi
- Department of Radiation Oncology, Stanford University School of Medicine
- Laure Aurelian
- Stanford University School of Medicine
- Matthew H. Porteus
- Department of Pediatrics, Stanford University School of Medicine
- DOI
- https://doi.org/10.1038/s41467-019-11962-8
- Journal volume & issue
-
Vol. 10,
no. 1
pp. 1 – 14
Abstract
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.
