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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
UCLA Medical Center, Santa Monica


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


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:

Joglekar AV, Stein L, Ho M, Hoban MD, Hollis RP, Kohn DB. Dissecting the Mechanism of Histone Deacetylase Inhibitors to Enhance the Activity of Zinc Finger Nucleases Delivered by Integrase-Defective Lentiviral Vectors. Hum Gene Ther. 2014 Apr 2. [Epub ahead of print]

Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther. 2014 Mar;22(3):607-22. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20.

De Oliveira SN, Ryan C, Giannoni F, Hardee CL, Tremcinska I, Katebian B, Wherley J, Sahaghian A, Tu A, Grogan T, Elashoff D, Cooper LJ, Hollis RP, Kohn DB. Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. Hum Gene Ther. 2013 Oct;24(10):824-39. doi: 10.1089/hum.2012.202.

Joglekar AV, Hollis RP, Kuftinec G, Senadheera S, Chan R, Kohn DB. Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Mol Ther. 2013 Sep;21(9):1705-17. doi: 10.1038/mt.2013.106. Epub 2013 Jul 16.

Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, Kempert P, Shupien S, Abdel-Azim H, Walters MC, Meiselman HJ, Wenby RB, Gruber T, Marder V, Coates TD, Kohn DB. Beta-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest. 2013 Jul 1. pii: 67930. doi: 10.1172/JCI67930. [Epub ahead of print]