Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Author:

Hoban Megan D.1ORCID,Cost Gregory J.2ORCID,Mendel Matthew C.2,Romero Zulema1ORCID,Kaufman Michael L.1ORCID,Joglekar Alok V.1,Ho Michelle1,Lumaquin Dianne1,Gray David1ORCID,Lill Georgia R.1ORCID,Cooper Aaron R.3ORCID,Urbinati Fabrizia1,Senadheera Shantha1,Zhu Allen2,Liu Pei-Qi2,Paschon David E.2,Zhang Lei2,Rebar Edward J.2,Wilber Andrew4,Wang Xiaoyan5,Gregory Philip D.2,Holmes Michael C.2,Reik Andreas2,Hollis Roger P.1,Kohn Donald B.16ORCID

Affiliation:

1. Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;

2. Sangamo BioSciences Inc., Richmond, CA;

3. Molecular Biology Interdepartmental PhD Program, University of California, Los Angeles, Los Angeles, CA;

4. Department of Medical Microbiology, Immunology and Cell Biology, Southern Illinois University School of Medicine, Springfield, IL;

5. Department of General Internal Medicine and Health Services Research, University of California, Los Angeles, Los Angeles, CA; and

6. Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA

Abstract

Key Points Delivery of ZFNs and donor templates results in high levels of gene correction in human CD34+ cells from multiple sources, including SCD BM. Modified CD34+ cells are capable of engrafting immunocompromised NSG mice and produce cells from multiple lineages.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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