Discovery of small molecule modifiers of microRNAs for the treatment of HCV infection.

While RNA has traditionally been viewed as a mechanism to transfer genetic information, its importance and functionality has only truly been demonstrated in the past 20 years. One prime example of this significance can be found within microRNAs (miRNAs), which are involved in the regulation of a substantial number of human genes. Consequently, these miRNAs represent a novel target for therapeutic agents towards the treatment of a variety of diseases and disorders. Specifically, misregulation of miR-122 has been demonstrated to be relevant in the cellular propagation of Hepatitis C (HCV), and thus modulators of miR-122 can have a substantial effect on viral loads. This protocol describes the development of an assay for the discovery of small molecule regulators of miR-122, and ultimately HCV therapeutics. Due to the excellent pharmacokinetic properties of small molecule libraries, these regulators represent a substantial advantage over traditional antisense agents.