Donald Kohn

Donald Barry Kohn

Professor, MIMG, University of California Los Angeles

Professor, Molecular and Medical Pharmacology, University of California Los Angeles

Professor, Pediatrics, University of California Los Angeles


BOX 957243, 3163 Terasaki SLB
Los Angeles, CA 90095

Office Address:
610 Charles E. Young Drive South
Los Angeles, CA 90095

Fax Number:


Co-Leader, Center for Translational Technologies
Member, Blood and Marrow Transplant Center, CTSI, Center for Duchenne Muscular Dystrophy, Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, Genetics & Genomics GPB Home Area, Immunity, Microbes & Molecular Pathogenesis GPB Home Area, Molecular Pharmacology GPB Home Area, Pilot and Collaborative Translational Clinical Studies Program, Tumor Immunology Program at the Jonsson Comprehensive Cancer Center (JCCC)

Research Interests

The research in the Kohn lab 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. Dr. 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. His laboratory is currently focused on three main project 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, 2) immunotherapy for cancer and leukemia by genetic modification of hematopoietic stem cells, and 3) the use of lentiviral vectors to direct the differentiation of human pluripotent stem cells for the production of pancreatic beta cells.


Donald B. Kohn, M.D. is Professor of Microbiology, Immunology and Molecular Genetics (MIMG) and Pediatrics, the Director of the UCLA Human Gene Medicine Program, and is a member of the Broad Stem Cell Research Center and the Jonsson Comprehensive Cancer Center. He joined the faculty of UCLA in 2009. He received a B.S in Biology and an M.S. in Microbiology from the University of Illinois-Urbana and his MD in 1982 at the University of Wisconsin-Madison. He performed Pediatric Internship and Residency at the University of Wisconsin Hospitals and then a Medical Staff Fellowship in the Metabolism Branch of the National Cancer Institute of the NIH. He was at Childrens Hospital Los Angeles, USC Keck School of Medicine for 21 years, where he rose to the rank of Professor and served as the Head of the Division of Research Immunology/Bone Marrow Transplantation from 2002-2009. His research program studies gene therapy using hematopoietic stem cells, performing basic studies of gene transfer and expression and translating these findings to clinical trials of gene therapy for congenital immune deficiencies and pediatric AIDS. He was the President of the American Society of Gene Therapy (2003-2004) and was the recipient of an Elizabeth Glaser Scientist Award from the Pediatric AIDS Foundation and a Distinguished Clinical Scientist Award from the Doris Duke Charitable Foundation.