Dr. Donald Kohn is a Professor in the Departments of Microbiology, Immunology & Molecular Genetics (M.I.M.G.) and Pediatrics and is the Director of the Human Gene Medicine Program. He served as an attending physician in the pediatric bone marrow transplant program at Childrens Hospital Los Angeles for more than 20 years. His research focuses on the development of new methods to treated genetic diseases of blood cells and cancer and leukemia by gene modification of hematopoietic stem cells. Kohn’s group was the first (and still the only one) to perform a clinical trial with gene transfer to umbilical cord blood CD34+ cells for a genetic disorder (ADA-SCID in 1993) and the first in the U.S. to initiate a clinical trial of gene therapy for pediatric AIDS using bone marrow stem cells.

Kohn's laboratory is currently focused on two main areas: 1) the development and clinical evaluation of improved methods for gene therapy of genetic diseases of blood cells targeting hematopoietic stem cells, especially ADA-deficient SCID and sickle cell disease, and 2) immunotherapy for cancer and leukemia by genetic modification of hematopoietic stem cells to produce targeted effector cells. Pre-clinical studies are focused on the use of lentiviral vectors for gene addition to stem cells and zinc-finger nucleases for gene correction by homologous recombination. His lab is currently performing a clinical trial of retroviral vector-mediated ADA gene transfer for children with ADA-deficient SCID and is working toward trials using lentiviral vectors for ADA-deficient SCID and sickle cell disease. In addition, Kohn’s group is participating as one of three U.S. sites for an NIAID-funded clinical trial of gene therapy for X-SCID.