RNA Interference: Molecular mechanism and milestone therapeutic applications

In time, we have become gradually more aware of the fact that RNAs play a vital role in regulation of gene expression. The discovery of RNA interference (RNAi) also called post transcriptional gene silencing (PTGS), gave a booster shot to the world of RNA and molecular biology, a set of reactions involving the sequence-specific degradation of targeted mRNA by short double-stranded RNA molecules. Swift progress in our knowledge of RNAi-based mechanisms has made us enabled to use this powerful molecular process in studies of gene action as well as in biological and therapeutic applications for the treatment of disease. RNAi has become a valuable research tool, both in cell culture and in living organisms, because synthetic dsRNA introduced into cells can selectively and vigorously induce suppression of specific genes of interest. RNAi may be used for large-scale screens that can systematically silent the specific gene, which can help to identify the components necessary for a particular cellular process or an event such as cell division. The pathway is also used as a practical tool in biotechnology and medicine. Currently there are various clinical trials involving RNAi, and there is an expectation for the list of new applications to grow at an exceptional rate. This article focuses on our modern understanding about the mechanisms and therapeutic applications of RNAi.