Scientific Interest(s):
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.
His laboratory is currently focused on three main project areas: 1) the development and clinical evaluation of improved methods for gene therapy of genetic diseases of blood cells targeting hematopoietic stem cells, 2) immunotherapy for cancer and leukemia by genetic modification of hematopoietic stem cells, and 3) the use of lentiviral vectors to direct the differentiation of human pluripotent stem cells for the production of pancreatic beta cells.
Selected Cancer-Related Publications:
Kohn DB, Candotti F. Gene therapy fulfilling its promise. N Engl J Med. 2009 Jan 29;360(5):518-21.
Carbonaro DA, Jin X, Cotoi D, Mi T, Yu XJ, Skelton DC, Dorey F, Kellems RE, Blackburn MR, Kohn DB. Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion. Blood. 2008 Jun 15;111(12):5745-54. Epub 2008 Mar 20.
Kahl CA, Cannon PM, Oldenburg J, Tarantal AF, Kohn DB. Tissue-specific restriction of cyclophilin A-independent HIV-1- and SIV-derived lentiviral vectors. Gene Ther. 2008 Aug;15(15):1079-89. Epub 2008 Apr 3.
Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, Hershfield MS, Candotti F, Kohn DB. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood. 2007 Jan 15;109(2):503-6. Epub 2006 Sep 14.
Blumenthal M, Skelton D, Pepper KA, Jahn T, Methangkool E, Kohn DB. Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Mol Ther. 2007 Jan;15(1):183-92.