Research in Human Gene Therapy
Bench to Bedside
The Program in Human Gene Therapy is dedicated to advancing gene transfer technologies and applying them to develop new treatments for genetic and acquired diseases. Our work spans the full research spectrum—from designing and testing novel vector systems to bringing the most promising approaches into clinical trials.
We focus on both viral and non-viral delivery platforms, evaluating their performance in relevant animal models and investigating the molecular mechanisms that drive vector transduction. This research informs the development of safer, more effective therapies.
Our major disease targets include hemophilia, hepatitis B and C infections, and diabetes. A second area of emphasis explores the role of small RNAs in mammalian gene regulation, providing new insights that may open additional therapeutic avenues.
Through this work, we aim to transform discoveries at the bench into life-changing therapies for patients worldwide.