Donald B. Kohn, M.D.

Donald B. Kohn, M.D. 

Distinguished Professor, Microbiology, Immunology and Molecular Genetics; Pediatrics (Hematology/Oncology); Molecular and Medical Pharmacology


Donald B. Kohn, M.D., studies the biology of blood stem cells, which are located in the bone marrow and have two important properties: they can duplicate themselves and they can create all types of blood cells. Over the course of 30 years of research, Kohn has developed new clinical methods to treat genetic blood diseases using blood stem cells that have been modified to remove genetic mutations.

Kohn’s blood stem cell gene therapy method collects some of a patient’s own blood stem cells and either adds a good copy of the defective gene or fixes the broken genes to eliminate disease-causing mutations. The patient then receives a transplant of their own corrected stem cells, which will ideally create an ongoing supply of healthy blood cells. Importantly, this method eliminates the risk of rejection associated with receiving a bone marrow transplant from a different person, meaning the patient doesn’t have to take a lifelong supply of anti-rejection drugs.

Kohn’s clinical trials for adenosine deaminase-deficient severe combined immunodeficiency (also known as ADA-SCID or bubble baby disease), a condition where babies are born without an immune system and often don’t survive past the first two years of life, have cured more than 50 babies to date. Babies with the condition and their families have traveled to UCLA for this life-saving treatment from as far away as Lebanon and a new company was formed in 2016 to further develop the therapy and make it available at other centers and to more patients.

Kohn is now applying similar blood stem cell gene therapy techniques in clinical trials for two other diseases. One of these diseases is X-linked chronic granulomatous disease, a rare inherited immunodeficiency disorder that prevents white blood cells from effectively killing foreign invaders such as bacteria, fungi or other microorganisms. If untreated, patients often succumb to chronic granulomatous disease within the first decades of life.

The second disease is sickle cell disease, the most common inherited blood disorder in the United States. This disease causes abnormal ‘sickle-shaped’ red blood cells that block small blood vessels and do not provide the appropriate amount of oxygen to the body, resulting in debilitating pain and organ damage. Kohn’s clinical trial seeks to overcome or repair the genetic mutation that causes this devastating disease, which impacts millions worldwide.

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 completed a pediatric internship and residency in Wisconsin followed by a medical staff fellowship in the Lymphoid Malignancies Branch (formerly the Metabolism Branch) of the National Cancer Institute.

Kohn began working on gene therapy as a fellow at the National Institutes of Health in 1985 and then began practicing as a pediatric bone marrow transplant physician at Children’s Hospital Los Angeles in 1987.  While practicing at Children’s Hospital Los Angeles, he started his own lab focused on stem cell research and has continued this work, advancing new therapies from the lab to the clinic.


Honors & Affiliations


  • Doris Duke Charitable Foundation Distinguished Clinical Scientist Award Recipient, 2000-2007
  • Elizabeth Glaser Scientist Award from the Pediatric AIDS Foundation, 1996-2001


  • President, Clinical Immunology Society, 2014
  • President, American Society of Gene and Cell Therapy, 2004
  • American Society for Gene and Cell Therapy
  • American Society of Hematology
  • American Society for Blood and Marrow Transplantation
  • Jonsson Comprehensive Cancer Center Tumor Immunology Program


Kohn’s work is funded by the California Institute for Regenerative Medicine, the National Institutes of Health, and the UCLA Broad Stem Cell Research Center, including support from the Hina Patel Foundation.


UCLA gene therapy trial for sickle cell disease:Evie’s story
For Evie Junior, living with sickle cell disease has been like running a marathon. “But it’s a marathon where as you keep going, the trail gets rockier and then you lose your shoes,” the 27-year-old said. “It gets harder as you get older. Things start to fail and all you can think about is how much worse it’s going to get down the road.”
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
New methods for the treatment of genetic blood diseases
Dr. Kohn's research focuses on the development of new methods to treat genetic blood diseases. In this video, he discusses a method that uses a patient's own blood-forming stem cells and a gene editing technique to correct genetic mutations that cause blood diseases, such as sickle cell and “bubble baby” disease.
Hussein's Lifesaving Stem Cell Treatment
When he was born in September 2015, Hussein El Kerdi looked like a healthy baby boy. No one knew that his immune cells lacked an important enzyme. But the absence of that enzyme would profoundly change the El Kerdi family’s life, sending them on a journey from their small hometown in Lebanon to UCLA. Their one goal: to save Hussein’s life with a stem cell gene therapy developed by Dr. Donald Kohn, a member of the UCLA Broad Stem Cell Research Center.
Dr. Kohn discusses his clinical trials for blood diseases
Dr. Donald Kohn, has dedicated his career to understanding the genetic mutations that cause blood disorders. As a pediatrician, Dr. Kohn is particularly interested in discovering treatments for blood diseases that begin in early childhood. His ultimate goal is to create stem cell gene therapy treatments that take a patient’s own blood-producing stem cells and fix the broken genes to eliminate the disease-causing mutations and the associated debilitating symptoms. This pioneering work gives hope to parents and families whose children suffer from genetic blood disorders, such as sickle cell disease. In this video, Dr. Kohn discusses his work and talks about the three clinical trials he is currently running.
UCLA Researchers Announce Gene Therapy Cure for ...
On November 18, 2014, researchers at UCLA announced that they had cured 18 children who were born with the so-called Bubble Baby disease, a genetic disorder that leaves the young sufferers without a working immune system, putting them at risk of death from infections, even the common cold. A team led by Dr. Donald Kohn, a stem cell researcher at the university’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research in Los Angeles, developed the breakthrough that cured 18 children who had adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).
Breakthroughs in Sickle Cell Disease Research
Doris Duke Charitable Foundation Director for Medical Research Sindy Escobar Alvarez, Ph.D., sat down with three of the country’s top sickle cell disease researchers to yield insights from the strides being made through their innovative research, examine the field’s most substantial challenges and discuss the most potent opportunities ahead. Hear from Daniel Bauer, M.D., Ph.D, hematologist at Boston Children’s Hospital and associate professor at Harvard Medical School; Donald B. Kohn, M.D., of University of California, Los Angeles; and Punam Malik, M.D., Marjorie Johnson Chair of Gene and Cell Therapy at Cincinnati Children’s Hospital, as they share what they have learned through their work in this disease area, why this is an opportune moment to accelerate curative therapies for sickle cell anemia and what the field needs to meet the moment.

Clinical Trials