Donald B. Kohn, MD

Donald B. Kohn, MD 



Donald Kohn, MD, focuses on the development of new methods to treat genetic diseases of blood cells, such as sickle cell and “bubble baby” disease, as well as using genetic modification of blood stem cells to treat cancer and leukemia. Kohn’s lab studies methods for effective gene transfer and expression and has translated those findings from the lab into clinical trials of gene therapy for congenital immune deficiencies and pediatric AIDS. A trial for sickle cell disease is currently under development.

Current projects include developing new approaches for gene therapy of ADA-deficient Severe Combined Immunodeficiency, the “bubble baby” disease, employing transfer of a normal ADA gene into blood stem cells, correction of the ADA gene within stem cells or direct administration of the ADA gene into the bloodstream. Parallel studies to treat sickle cell disease are underway, either by adding a normal beta-globin gene to a patient’s bone marrow stem cells or by correcting the sickle globin gene itself. Dr. Kohn’s lab is part of a large collaborative group at UCLA working on new ways to treat cancer and leukemia by harnessing a patient’s immune cells to fight the disease. His component of this multi-disciplinary team is focused on modifying the blood stem cells to produce white blood cells that recognize and kill cancer and leukemia cells.

Dr. Kohn has focused on stem cell research since he started his own lab in 1987. A major element of his work has been the study of the basic biology of blood stem cells in order to understand how to isolate, genetically modify and re-transplant them back into people. His research group also has developed several of the methods used to transfer genes into human stem cells. His clinical work in pediatric bone marrow transplant also involves stem cells, as they are the key cells in bone marrow that produce the effects of transplantation. He hopes his research can continue to provide clinical benefits to patients with immune disorders, blood cell diseases and cancer and leukemia.

In addition to being a member of the Broad Stem Cell Research Center, Dr. Kohn directs the Human Gene Medicine Program at UCLA and is a member of UCLA’s Jonsson Comprehensive Cancer Center. He also served as president of the American Society of Gene Therapy from 2003 to 2004, and currently is on the Board of Councilors for the Clinical Immunology Society. He has been the recipient of the Elizabeth Glaser Scientist Award from the Pediatric AIDS Foundation and a Distinguished Clinical Scientist Award from the Doris Duke Charitable Foundation.

A professor of pediatrics and microbiology, immunology and molecular genetics in the life sciences, Kohn was recruited to the UCLA faculty in 2009 from Childrens Hospital Los Angeles, where he served as an attending physician in the pediatric bone marrow transplant program for more than 20 years. Dr. Kohn earned his bachelor’s and master’s degrees from the University of Illinois, Champaign-Urbana and his medical degree from the University of Wisconsin School of Medicine. He did a pediatric internship and residency in Wisconsin and then completed a medical staff fellowship in the Metabolism Branch of the National Cancer Institute.

Dr. Kohn’s work is currently funded by the California Institute of Regenerative Medicine, the National Institutes of Health, the Doris Duke Charitable Foundation and the Juvenile Diabetes Research Foundation, with past funding also from the March of Dimes, the American Cancer Society and the Leukemia and Lymphoma Society. His lab is also a site for active training of UCLA undergraduate and graduate students and post-doctoral fellows.


Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency
Published March 27, 2017 - The Journal of Clinical Investigation

Correction of the sickle-cell disease mutation in human hematopoietic stem/progenitor cells
Published Online March 2015 - Blood Journal

T-Cell Immunotherapy: Looking Forward
September 2014 - Molecular Therapy



Let There Be Tiny Miracles
Celebrating the launch of The Centennial Campaign for UCLA, this video offers a glimpse into the miraculous work of Broad Stem Cell Research Center faculty member Dr. Donald B. Kohn and his impactful research to cure blood diseases.
Dr. Donald Kohn Discusses Stem Cell Research Breakthrough
Dr. Donald Kohn discusses a new breakthrough in stem cell research for sickle cell disease, and translating basic science discoveries to treatment.
Bone Marrow Stem Cells and Gene Therapy
UCLA Life Sciences Public Lecture: "Bone Marrow Stem Cells: Developing New Therapies in the Fight Against Disease" Donald Kohn, M.D. January 18, 2011