Biography
Donald B. Kohn, M.D. is a Professor in the Departments of Pediatrics and Molecular Microbiology & Immunology at the U.S.C. Keck School of Medicine. He is an Attending Physician in Bone Marrow Transplantation at Childrens Hospital Los Angeles, where he also is the Director of the John Connell Gene Therapy Program. He runs a research program studying methods to transfer genes into blood stem cells and has performed pioneering clinical studies of gene therapy for children with severe combined immune deficiency (bubble baby disease) and AIDS. He is also performing research on methods to induce the immune system to eliminate leukemia.
Donald Kohn attended the University of Illinois-Urbana and received a B.S. in Biology in 1976 and an M.S. in Microbiology in 1978. He received his MD at the University of Wisconsin-Madison Medical School in 1982. He was an Intern and Resident in Pediatrics at the University of Wisconsin Hospitals from 1982-1985. Following a Fellowship in Immunology with Dr. R. Michael Blaese in the Metabolism Branch of the National Cancer Institute at the National Institutes of Health, Bethesda from 1985-1987, Dr. Kohn became an Attending Physician on the Bone Marrow Transplantation service at Childrens Hospital Los Angeles. Dr Kohn has been a professor at the University of Southern California School of Medicine in the Departments of Pediatrics and Microbiology since 1977.
Abstract
Gene Therapy for Blood Diseases Using Hematopoietic Stem Cells
Gene therapy is an emerging medical modality in which genetic diseases will be corrected by transfer of a normal version of the relevant gene into a patient’s cells. Gene transfer into the hematopoietic stem cells (HSC) from the bone marrow of patients with blood diseases, such as sickle cell anemia, and immune deficiencies, such as "bubble baby" disease, could provide a safer treatment than the current use of bone marrow from another person. While the use of gene therapy to treat blood diseases is logical, this vision remains unfulfilled, primarily due to the inadequacies of the tools used for gene transfer and expression. Over the past decade, continued research has led to steady improvements in the available techniques for gene transfer, gene expression, and hematopoietic stem cell manipulation.
Dr. Kohn’s studies will be to continue this process, by:
- Performing basic laboratory research to improve these tools further;
- Implementing clinical trials to evaluate the efficacy of the gene therapy techniques; and
- Mentoring new physician/scientists on the science of gene therapy and the proper development and performance of clinical research.