Dr. Donald B. Kohn, MD, is an American pediatric physician-scientist whose pioneering work in gene therapy has helped create new treatment possibilities for children affected by serious genetic and immune-system disorders.
Based at the University of California, Los Angeles (UCLA), he has spent nearly four decades developing therapies that use patients’ own blood-forming stem cells to address the underlying causes of disease.
His work represents an important connection between laboratory science and practical medical treatment, particularly for children who previously had very limited therapeutic options.
Education and Professional Career
Dr. Kohn completed his medical education and specialized training in pediatrics and pediatric bone marrow transplantation before developing a strong research focus on genetic diseases and immune-system disorders.
He joined UCLA in 2009 and became a distinguished professor of microbiology, immunology and molecular genetics, as well as pediatrics and molecular and medical pharmacology.
He is also associated with the UCLA Broad Stem Cell Research Center, where researchers investigate new approaches to treating diseases using stem cells and genetic technologies.
His clinical experience treating children who needed bone marrow transplants helped inspire his interest in developing alternative treatments that could avoid some of the complications associated with donor transplantation.
Transforming Treatment Through Gene Therapy
One of Dr. Kohn’s most significant contributions involves adenosine deaminase-deficient severe combined immunodeficiency, commonly known as ADA-SCID.
This rare genetic condition prevents the immune system from functioning properly, leaving affected children highly vulnerable to serious infections.
Dr. Kohn and his collaborators began treating patients with gene therapy for ADA-SCID in 1993. Their approach uses a patient’s own blood-forming stem cells, introducing a healthy copy of the gene responsible for producing the missing enzyme.
The goal is to allow the patient’s body to develop functioning immune cells without relying on a donor transplant.
Long-term results published in 2025 from a major study showed that immune function was restored and maintained in 59 of 62 children treated with this approach.
A Major Medical Milestone in 2026
Another important achievement came in March 2026, when the U.S. Food and Drug Administration approved Kresladi, a gene therapy for severe leukocyte adhesion deficiency type I (LAD-I).
Dr. Kohn led UCLA’s participation in the multicenter clinical trial supporting this treatment. The therapy is designed to address the underlying genetic defect responsible for the disorder.
All nine children in the clinical trial survived and demonstrated sustained restoration of immune function. The approval represented a significant milestone for families affected by this rare and potentially life-threatening condition.
His laboratory continues investigating gene therapy and gene-editing approaches for other diseases, including sickle cell disease, alpha thalassemia and inherited immune deficiencies.
National Recognition and Continued Innovation
In October 2026, Dr. Kohn was elected to the National Academy of Medicine, one of the most prestigious honors in American medicine. The recognition reflects his substantial contributions to gene therapy and the advancement of treatments for serious childhood diseases.
He has also held leadership positions in the American Society of Gene and Cell Therapy and the Clinical Immunology Society.
Through decades of research, collaboration and clinical innovation, Dr. Donald Kohn has helped bring promising genetic treatments closer to the patients who need them.
His work continues to demonstrate how advances in gene therapy can move beyond scientific theory and become practical medical solutions, offering new hope to children and families facing challenging genetic conditions.
