Approaches for the Discovery of Small Molecule Ligands Targeting microRNAs

Abstract

RNA is essential for life, serving as the intermediate between genomic storage and protein function. As our knowledge of biological systems has grown, so has our understanding of RNA, revealing additional functions of this critical class of biomolecules. One class of RNA, microRNAs (miRNA), highlights the fundamental role of non-coding RNA in higher organisms. miRNAs regulate nearly every biological pathway through targeted translational suppression, and dysregulation of miRNA expression has been implicated in many human disease states. Thus, therapeutically targeting miRNAs with small molecule ligands is of growing importance. Herein we focus on methods employed to discover small molecule miRNA ligands, their successes thus far, and future directions for the field.