Our lab is focused on developing novel gene therapy approaches for inherited and acquired neurodegenerative disorders. Currently we are focusing on GM2-gangliosidoses (Tay-Sachs, Sandhoff diseases and AB variant) and use adeno-associated virus vector (AAV) system as a tool for gene transfer to the central and peripheral nervous system. Our research has already shown very encouraging results in a mouse model increasing the survival to almost 3 fold for this fatal disease. We are in process of testing multiple strategies to optimize the conditions with a target for clinical translation.
Our future directions include:
1) Understanding the basic biology of GM2 ganglioside and its effect on immune system; 2) Developing novel AAV vectors with in-built safety system;
3) Developing assays for hexosaminidase activity and peripheral blood markers for GM2 gangliosidoses; and
4) Understanding the inflammatory changes at gene expression and epigenetic level with and without AAV gene therapy treatment in GM2 gangliosidoses.My clinical research involves study of genetics, epigenetics, metagenomics and metabolomics of Autism. I collaborate on many studies for inherited metabolic disorders.