RNAi mechanisms and applications

Within the past two decades we have become increasingly aware of the roles that RNAs play in regulation of gene expression. The RNA world vas given a booster shot with the discovery of RNA interference (RNAi), a compendium of mechanisms involving small RNAs (less than 30 bases long) that regulate the expression of genes in a variety of eukaryotic organisms. Rapid progress in our understanding of RNAi-based mechanisms hits led to applications of this powerful process in studies of gene function as well as in therapeutic applications for the treatment of disease. RNAi-based therapies involve two-dimensional drug designs using only identification of good Watson-Crick base pairing between the RNAi guide strand and the tat-get, thereby resulting in rapid design and testing of RNAi triggers. To date there are several clinical trials using RNAi, and we should expect the list of ne v applications to grow, tit it phenomenal rate. This article summarizes our current knowledge about the mechanisms and applications of RNAi.