Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing-Reference-Cited by-同舟云学术

Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing

Published:2018-06-28 Issue:26 Volume:131 Page:2915-2928
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Li Chang¹,Psatha Nikoletta²,Sova Pavel¹,Gil Sucheol¹,Wang Hongjie¹,Kim Jiho¹,Kulkarni Chandana¹,Valensisi Cristina¹,Hawkins R. David¹,Stamatoyannopoulos George¹,Lieber André¹³

Affiliation:

1. Division of Medical Genetics, Department of Medicine,

2. Division of Hematology, Department of Medicine, and

3. Department of Pathology, University of Washington, Seattle, WA

Abstract

Key Points CRISPR/Cas9-mediated disruption of a BCL11A binding site in HSCs of β-YAC mice results in the reactivation of γ-globin in erythrocytes. Our approach for in vivo HSC genome editing that does not require HSC transplantation and myeloablation should simplify HSC gene therapy.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/131/26/2915/1466305/blood838540.pdf

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