Donald Kohn, MD

Los Angeles, California,
Over the course of 30 years of research, Dr. Kohn has developed new clinical methods to treat genetic blood diseases using blood stem cells that have been modified to remove genetic mutations.
Dr. 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.
Dr. 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. If untreated, patients often succumb to chronic granulomatous disease within the first decades of life. The second disease is sickle cell disease.
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 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.
Speaking In
3:00 PM - 4:00 PM (PDT)
Wednesday, June 15
Recent successes in genetic medicine (regulatory approvals of Zolgensma and Luxturna, for example)…
10:15 AM - 11:15 AM (PDT)
Thursday, June 16
During recent years, a few challenges and risks to patients have been reported emerging from viral…