Molecular Genetics and Therapeutics Lab is focused on developing and translating novel therapies for clinical applications. Towards this, we aim to define, characterize and overcome existing barriers by:
· The study of the biology and host response to gene delivery strategies.
· The development of enabling molecular technologies.
· The validation and translation of specific diagnostic and therapeutic programs.
The most important component of gene therapy in humans is the choice of an efficient and safe gene delivery system, otherwise known as vectors. Adeno-associated virus (AAV) based vectors have emerged as important tools for this purpose. However limitations, most notably, the immunological barriers related to the recipient and the virus coat protein preclude the universal application of this promising vector system.
Unraveling how host cells perceive the viral intrusion and their immediate and long-lived response to this infection process is thus crucial. Our laboratory's research interests traverse and investigate aspects of the virus-host 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. The information thus gathered is then used to bioengineer the vectors and develop improved gene delivery strategies. Such a multi-pronged basic biology approach to develop efficient vectors and their subsequent validation in pre-clinical models augurs well for their translation to human gene therapy.
Based on this approach, we have developed novel gene therapy strategies in pre-clinical models of hemophilia, ocular disorders such as Leber congenital amarousis and for molecular therapy of cancers such as hepatocellular carcinoma, myeloid leukemia and T-cell lymphoma.
Highlights of our research findings (Graphical abstract):
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- Congratulations to Ms Nusrat Khan for the ‘Outstanding Poster Presentation Award’ in the "23rd Annual virtual Meeting of the American Society of Gene and Cell therapy, May 12-15, 2020."
- Congratulations to Nusrat Khan, a Ph.D. student from lab for the American Society of Hematology abstract achievement award to present in the "61st American Society of Hematology meeting in Orlando, USA between December 7-10, 2019.">
- Shubham Maurya, a Ph.D. student from lab has been selected to deliver a poster presentation in the "ESGCT 27th Annual Congress in collaboration with SETGyC in Barcelona, Spain between October 22-25, 2019."
- Shubham Maurya, a Ph.D. student from lab has been selected to deliver a platform presentation in the "XXVII Congress of the International Society of Thrombosis and Hemostasis and 65th Annual Scientific and Standardization Committee (SSC) Meeting" held in Melbourne, Australia from July 6 – 10, 2019.