MicroRNA therapeutics: Discovering novel targets and developing specific therapy

Perspect Clin Res. Apr-Jun 2016;7(2):68-74. doi: 10.4103/2229-3485.179431.


MicroRNAs (miRNAs) are small non-coding RNA molecules that regulate gene expression in diverse biological process. They act as intracellular mediators that are necessary for various biological processes. MicroRNAs targeting pathways of human disease provide a new and potential powerful candidate for therapeutic intervention against various pathological conditions. Even though, the information about miRNA biology has significantly enriched but we still do not completely understand the mechanism of miRNA gene regulation. Various groups across the globe and pharmaceutical companies are conducting research and developments to explore miRNA based therapy and build a whole new area of miroRNA therapeutics. Consequently, few miRNAs have entered the preclinical and clinical stage and soon might be available in the market for use in humans.

Keywords: Clinical stage; gene expression; microRNA; therapeutics.

Publication types

  • Review