The research in the Kohn lab focuses on the development of new methods to treated genetic diseases of blood cells by gene modification of hematopoietic stem cells. Dr. 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 clinical trials of gene therapy for pediatric HIV/AIDS using bone marrow stem cells. Current projects include (1) performing clinical trials for ADA-deficient SCID, XSCID, Sickle Cell Disease and X-linked Chronic Granulomatous Disease, (2) developing new lentiviral vectors for regulated gene expression, (3) advancing techniques for site-specific gene editing for sickle cell disease and primary immune deficiency diseases, and (4) improving clinical hematopoietic stem cell processing. Our research group spans the bench-to-bedside spectrum and provides an excellent training environment for physician/scientists.
