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Abstract

microRNAs are endogenous small non-coding RNAs that regulate gene expression by interfering with translation or stability of target transcripts. The importance and varied functions of microRNAs are illustrated by the diverse phenotypes, including disease, that arise when microRNAs are mutated or improperly expressed. The association of microRNA dysfunction with disease phenotypes has given rise to the idea that selective modulation of microRNAs could alter the course of disease. With the recent demonstration that inhibition of miR-122 reduces viral load in HCV-infected chimpanzees, microRNA modulators are no longer merely theoretical, but have become strong candidate therapeutics. Here we review the evidence for microRNA dysfunction in human disease, as well as recent examples of microRNA modulation that provided therapeutic benefit.