Biogenesis and function of endogenous and exogenous siRNAs.

ABSTRACT

RNA interference (RNAi) is a sequence-specific gene silencing, or 'knockdown', mechanism facilitated by short duplex strands of RNA with sequence complementarity to target mRNAs. RNAi has many different forms, including posttranscriptional gene silencing (PTGS), and transcriptional gene silencing (TGS). Here, we review the biogenesis and function of an endogenous set of small RNA gene regulators, called microRNAs, as well as the mechanism of exogenously delivered small interfering RNAs. The potential applications of RNAi-based therapeutics are also highlighted.