Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

Author:

Peterson Christopher W.1,Wang Jianbin2,Norman Krystin K.1,Norgaard Zachary K.1,Humbert Olivier1,Tse Collette K.2,Yan Jenny J.2,Trimble Richard G.2,Shivak David A.2,Rebar Edward J.2,Gregory Philip D.2,Holmes Michael C.2,Kiem Hans-Peter134

Affiliation:

1. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA;

2. Sangamo BioSciences, Richmond, CA; and

3. Department of Medicine and

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

Abstract

Key Points This study is the first to show that genome-editing approaches can modify multilineage, long-term repopulating cells in a large animal model. We demonstrate that the persistence of genome-edited hematopoietic stem cells can be tracked in vivo in a mutation-specific manner.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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