Nucleic acid therapeutics
RNAi molecules can silence virtually any gene with high efficiency and specificity, highlighting its potential to treat disease.
The research area
Downregulating a disease-causing gene in a sequence-specific manner has a profound impact on mitigating human diseases conditions. Traditionally this is achieved by targeting the mRNA, the fundamental cellular machinery that creates protein using antisense technology or by using interfering RNA (RNAi technology).
RNAi is an endogenous pathway for the post-transcriptional silencing of gene expression that is triggered by double-stranded RNA (dsRNA), including endogenous microRNA (miRNA) and synthetic short interfering RNA (siRNA). By this pathway, RNAi molecules can silence the expression of virtually any gene with high efficiency and specificity, highlighting its potential for treating several diseases. Though researchers around the world have made significant achievements in understanding how these RNA based systems work, there are several challenges to overcome, both at the fundamental level as well as at the application level.
Our research group is focused on these aspects by employing engineering tools that interfere with the intracellular enzymatic processes and biological function.