Donald Kohn

Donald Kohn is a Professor at the University of California Los Angeles with joint appointments in the departments of Microbiology, Immunology & Molecular Genetics, Molecular and Medical Pharmacology, and Pediatrics. His research focuses on developing gene therapies for primary immune deficiencies and genetic blood disorders, particularly through hematopoietic stem cell engineering.

His primary research interests include gene therapy for severe combined immunodeficiencies (SCID), sickle cell disease, and leukocyte adhesion deficiencies. He develops novel lentiviral and CRISPR-based approaches to correct genetic defects while ensuring therapeutic safety and efficacy.

Kohn's recent work demonstrates significant advances in clinical gene therapy, including landmark studies on ADA-SCID and X-linked SCID treatments. His research portfolio shows increasing focus on precision genome editing techniques and improving long-term outcomes of hematopoietic stem cell therapies.

Current investigations explore base editing, CAR-T cell development from iPSCs, and optimized vector designs for hemoglobinopathies. His laboratory maintains active collaborations with multiple pediatric research centers to translate these discoveries into clinical applications.