The discovery of small RNA molecules as regulators of posttranscriptional gene silencing has paved the way to specifically target any given protein via the RNA interference (RNAi) pathway. An endogenous class of these molecules, the microRNA (miRNA), is proposed to control expression of up to one third of all genes and may be utilized as diagnostic and prognostic marker for diseases. In addition the recent employment of antagomirs that specifically inhibit function of a given miRNA represents a powerful tool to determine the role of these molecules in disease pathogenesis. Here, we describe our current understanding of the structure, biogenesis and function of small RNA, as well as their potential and limitation as novel therapeutic approaches.
Pharmacology & Therapeutics Vol. 117, Issue 1, p. 94-104