A review of currently identified small molecule modulators of microRNA function

microRNAs are small, noncoding RNA that negatively regulate gene expression. Since their discovery in 1993, approximately 2500 human mature microRNAs have been discovered and details of their biogenesis, mechanism of action, and function has been studied. Aberrant expression of microRNAs has since been observed in numerous disease states particularly cancer, neurologic disorders, autoimmune diseases, metabolic diseases and cardiovascular diseases. Because of this, a strong interest in developing novel therapies that modulate microRNA function has emerged. Although, several strategies have been employed, small molecule drugs have shown great promise due their inherent stability, bioavailability, and cost-efficiency. In this review, we discuss the microRNA modulating small molecules that have thus far been identified in the literature and highlight the need for continued research in this field. [Display omitted] •microRNAs are small, noncoding RNAs that post-transcriptionally regulate gene expression by binding the 3’ UTR of mRNA.•Aberrant expression of specific microRNAs has been observed in many diseases including cancer and neurological diseases.•microRNAs have been perceived as “undruggable”, however, research over the past decade has proven otherwise.•Small molecule modulators of microRNA have been discovered either through high-throughput screening or structural approaches.•Continued research will greatly improve our understanding of small molecule drug design for these unique targets.