Donald Barry Kohn

Donald B. Kohn is a Professor of Molecular and Medical Pharmacology, Pediatrics, and Medicine at the University of California, Los Angeles (UCLA). He holds joint appointments across departments within the School of Medicine, focusing on gene therapy, hematopoietic stem cell research, and the treatment of genetic disorders such as severe combined immunodeficiency (SCID) and sickle cell disease. His work spans clinical trials, translational research, and translational medicine, with a strong emphasis on advancing lentiviral vector technologies and ensuring their safety and efficacy.

• Affiliations: UCLA Medical Network, Primary Immune Deficiency Treatment Consortium (PIDTC), and various NIH-funded initiatives.
• Research Focus: Gene therapy for primary immunodeficiencies, hematopoietic stem cell transplantation, CRISPR/Cas9 and base editing applications in blood disorders, and managing long-term outcomes of gene therapy patients.

Dr. Kohn's research interests include optimizing lentiviral vectors, understanding insertional oncogenesis risks, and developing curative therapies for conditions like ADA-SCID, X-linked chronic granulomatous disease (CGD), and sickle cell anemia. He collaborates extensively on clinical trials, including Phase I/II studies for gene therapy in pediatric patients and translational studies on CAR-T and NKT cell therapies derived from hematopoietic stem cells.

His team has contributed to landmark studies on immune reconstitution post-transplantation, the efficacy of gene therapy in newborns screened for SCID, and strategies to mitigate adverse effects such as myelodysplasia. Key projects include the design of self-inactivating lentiviral vectors, CRISPR-based editing of hematopoietic stem cells, and the development of scalable gene therapy protocols for widespread clinical application.