Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates-Reference-Cited by-同舟云学术

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

Published:2019-07-31 Issue:503 Volume:11 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Humbert Olivier¹^ORCID,Radtke Stefan¹^ORCID,Samuelson Clare¹,Carrillo Ray R.¹,Perez Anai M.¹,Reddy Sowmya S.¹,Lux Christopher²^ORCID,Pattabhi Sowmya²^ORCID,Schefter Lauren E.¹,Negre Olivier³,Lee Ciaran M.⁴⁵^ORCID,Bao Gang⁴,Adair Jennifer E.¹⁶^ORCID,Peterson Christopher W.¹^ORCID,Rawlings David J.²⁷⁸^ORCID,Scharenberg Andrew M.²⁷⁸⁹,Kiem Hans-Peter¹⁶^ORCID

Affiliation:

1. Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.

2. Seattle Children’s Research Institute, Seattle, WA 98101, USA.

3. Bluebird Bio Inc., Cambridge, MA 02142, USA.

4. Department of Bioengineering, Rice University, Houston, TX 77251, USA.

5. Alimentary Pharmabiotic Centre Microbiome Ireland, University College Cork, Cork T12 K8AF, Ireland.

6. Department of Medicine, University of Washington, Seattle, WA 98195, USA.

7. Department of Pediatrics, University of Washington, Seattle, WA 98195, USA.

8. Department of Immunology, University of Washington, Seattle, WA 98195, USA.

9. Casebia Therapeutics, Cambridge, MA 02139, USA.

Abstract

CRISPR-Cas9–edited hematopoietic stem cells produce long-term engraftment and fetal hemoglobin reactivation in nonhuman primates.