title: Small molecule modifiers of the microRNA and RNA interference pathway.
abstract: Recently, the RNA interference (RNAi) pathway has become the target of small molecule inhibitors and activators. RNAi has been well established as a research tool in the sequence-specific silencing of genes in eukaryotic cells and organisms by using exogenous, small, double-stranded RNA molecules of approximately 20 nucleotides. Moreover, a recently discovered post-transcriptional gene regulatory mechanism employs microRNAs (miRNAs), a class of endogenously expressed small RNA molecules, which are processed via the RNAi pathway. The chemical modulation of RNAi has important therapeutic relevance, because a wide range of miRNAs has been linked to a variety of human diseases, especially cancer. Thus, the activation of tumor-suppressive miRNAs and the inhibition of oncogenic miRNAs by small molecules have the potential to provide a fundamentally new approach for the development of cancer therapeutics.
authors: Department of Chemistry, North Carolina State University, Raleigh, North Carolina 27695-8204, USA. alex_deiters@ncsu.edu
authors: Alexander
authors: A
authors: Deiters
mesh_terms: D000818:Animals; D006801:Humans; D035683:MicroRNAs / Q000037:antagonists & inhibitors* / Q000096:biosynthesis; D034622:RNA Interference / Q000187:drug effects*; D034741:RNA, Small Interfering / Q000096:biosynthesis
chemical_list: C524940:MIRN21 microRNA, human; D035683:MicroRNAs; D034741:RNA, Small Interfering