BE Seminar: Indigenous technologies for Gene therapy: Progress and Prospects

Title: Indigenous technologies for Gene therapy: Progress and Prospects

Speaker: Dr. Jayandharan Rao,

Abstract: The most important component of gene therapy in humans is the choice of an efficient and safe gene delivery system, otherwise known as vectors. Viruses have emerged as important tools for this purpose. This is due to their natural ability to target specific tissues in humans. Several limitations, most notably the immunological barriers related to the recipient and the virus shell, preclude the universal application of viral vectors such as AAV. Unravelling how host cells perceive viral intrusion and their immediate and long-lived response to this process is thus crucial. Our idea is to traverse and investigate aspects of the virus life cycle, dissect the tug-of-war initiated between these players to establish primacy, and dwell on how it is possible to alter this milieu in favor of the virus to generate improved gene delivery strategies. Dr.Jayandharan believes that this multi-pronged and comprehensive approach to develop an AAV-based vector system encompassing the basic biology of the virus and pre-clinical validation in different disease models [Leber congenital amarousis, muscular dystrophy, diabetes, etc.] augurs well for their potential use in humans. Based on these concepts, we have developed highly efficient AAV-based gene therapy approaches for their potential therapeutic use. Their lab routinely tests some of the exciting leads gathered by our understanding of AAV-host interactions in preclinical models of genetic disorders.

About The Speaker: Prof. Rao is currently a Professor in IIT Kanpur. His group specializes in gene therapy. He is working on fostering equitable access for gene and cell therapy products around the world.