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Donald B. Kohn, M.D.
Donald B. Kohn, M.D.


Pediatric Hematopoietic Stem Cell Transplantation

General Information:



Professor, Department of Microbiology, Immunology, and Molecular Genetics, Department of Pediatrics
Member, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research
Member, JCCC Tumor Immunology Program Area

Hospital Affiliation(s):

Ronald Reagan UCLA Medical Center
Santa Monica-UCLA Medical Center and Orthopaedic Hospital


Allergy and Immunology, National Cancer Institute, 1985 - 1987
Pediatrics, University of Wisconsin Hospitals and Clinics, 1983 - 1985
Pediatrics, University of Wisconsin Hospitals and Clinics, 1982 - 1983
Medical Degree:
M.D., University of Wisconsin Medical School, 1982
M.S., Microbiology, University of Illinois-Urbana, 1978
B.S., Biology, University of Illinois-Urbana, 1976


Medical Board Certification(s):
Pediatrics, American Board of Pediatrics, 1988

Contact Information:

(310) 794-1964

Practice Information:

Clinical Interest(s):
Genetic Diseases of Blood Cells
Pediatric Leukemia

Scientific Interest(s):

Dr. Donald Kohn is a Professor in the Departments of Microbiology, Immunology & Molecular Genetics (M.I.M.G.) and Pediatrics and is the Director of the Human Gene Medicine Program. He served as an attending physician in the pediatric bone marrow transplant program at Childrens Hospital Los Angeles for more than 20 years. His research focuses on the development of new methods to treated genetic diseases of blood cells and cancer and leukemia by gene modification of hematopoietic stem cells. Kohn’s group was the first (and still the only one) to perform a clinical trial with gene transfer to umbilical cord blood CD34+ cells for a genetic disorder (ADA-SCID in 1993) and the first in the U.S. to initiate a clinical trial of gene therapy for pediatric AIDS using bone marrow stem cells.

Kohn's laboratory is currently focused on two main areas: 1) the development and clinical evaluation of improved methods for gene therapy of genetic diseases of blood cells targeting hematopoietic stem cells, especially ADA-deficient SCID and sickle cell disease, and 2) immunotherapy for cancer and leukemia by genetic modification of hematopoietic stem cells to produce targeted effector cells. Pre-clinical studies are focused on the use of lentiviral vectors for gene addition to stem cells and zinc-finger nucleases for gene correction by homologous recombination. His lab is currently performing a clinical trial of retroviral vector-mediated ADA gene transfer for children with ADA-deficient SCID and is working toward trials using lentiviral vectors for ADA-deficient SCID and sickle cell disease. In addition, Kohn’s group is participating as one of three U.S. sites for an NIAID-funded clinical trial of gene therapy for X-SCID.

Selected Cancer-Related Publications:

Tarantal AF, Giannoni F, Lee CC, Wherley J, Sumiyoshi T, Martinez M, Kahl CA, Elashoff D, Louie SG, Kohn DB. Nonmyeloablative conditioning regimen to increase engraftment of gene-modified hematopoietic stem cells in young rhesus monkeys. Mol Ther. 2012 May;20(5):1033-45. doi: 10.1038/mt.2011.312. Epub 2012 Jan 31.

Corrigan-Curay J, Cohen-Haguenauer O, O'Reilly M, Ross SR, Fan H, Rosenberg N, Somia N, King N, Friedmann T, Dunbar C, Aiuti A, Naldini L, Baum C, von Kalle C, Kiem HP, Montini E, Bushman F, Sorrentino BP, Carrondo M, Malech H, Gahrton G, Shapiro R, Wolff L, Rosenthal E, Jambou R, Zaia J, Kohn DB. Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther. 2012 Jun;20(6):1084-94.

Vatakis DN, Koya RC, Nixon CC, Wei L, Kim SG, Avancena P, Bristol G, Baltimore D, Kohn DB, Ribas A, Radu CG, Galic Z, Zack JA. Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proc Natl Acad Sci U S A. 2011 Dec 20;108(51):E1408-16. Epub 2011 Nov 28.

Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol. 2010 Aug;28(8):839-47. Epub 2010 Jul 2.

Kohn DB, Candotti F. Gene therapy fulfilling its promise. N Engl J Med. 2009 Jan 29;360(5):518-21.