The Division of Human Gene Therapy at Stanford's School of Medicine is dedicated to advancing the science and clinical application of gene transfer technologies. Our mission is to develop transformative therapies for both genetic and acquired diseases through innovation in vector design, delivery systems, and translational research. 

Our work focuses on improving viral and non-viral vector systems, optimizing delivery methods, and understanding the molecular mechanisms that govern vector transduction. Using relevant preclinical models, we evaluate the most promising stategies for advancement into clinical trials. 

Primary disease targets include hemophilia, hepatitis B and C, and diabetes. We also investigate how small RNAs regulate gene expression in mammilian systems, with the aim of discovering novel therapeutic pathways. 

The Division is led by Dr. Mark Kay, M.D, PhD., and brings together scientists, clinicians, and trainees working at the intersection of basic research and clinical innovation.