RNA interference: A futuristic tool and its therapeutic applications

Abstract

RNA interference is a post transcriptional gene silencing mechanism that is triggered by double-stranded RNA (dsRNA). Various attributes of the 3′ end structure, including overhang length and sequence composition, plays a primary role in determining the position of the Dicer cleavage in both dsRNA and unimolecular, short hairpin RNA (shRNA). The specificity and robustness of RNAi have triggered an immense interest in using RNAi as a tool in various settings. RNAi is a mechanism for controlling normal gene expression which has recently began to be employed as a potential therapeutic agent for a wide range of disorders, including cancer, infectious diseases and metabolic disorders. Clinical trials with RNAi have now begin, but major obstacles, such as off-target effects, toxicity and unsafe delivery methods, have to be overcome before RNAi can be considered as a conventional drug. It is also used as a tool to improve crops by providing resistance against parasites and modified versions of siRNA that are directed against disease causing genes are being developed, some of which are already tested in clinical trials. In this paper, we first reviewed the RNAi mechanism and then focussed on some of its applications in biomedical research such as treatment for HIV, viral hepatitis and several other diseases.