Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development

Author:

Cannon Paula1,Asokan Aravind2,Czechowicz Agnieszka3,Hammond Paula4,Kohn Donald B.5,Lieber Andre6,Malik Punam7,Marks Peter8,Porteus Matthew3,Verhoeyen Els910,Weissman Drew11,Weissman Irving12,Kiem Hans-Peter13

Affiliation:

1. Department of Molecular Microbiology and Immunology, Keck School of Medicine, University of Southern California, Los Angeles, California, USA

2. Department of Surgery, Duke University School of Medicine, Durham, North Carolina, USA

3. Department of Pediatrics

4. Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA

5. Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA

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

7. Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA

8. U.S. Food and Drug Administration, Silver Spring, Maryland, USA

9. CIRI, Université de Lyon, INSERM, CNRS, ENS de Lyon, Lyon, France

10. Université de Nice, Nice, France

11. Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA

12. Stanford Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, California, USA

13. Clinical Research Division, Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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