title: MicroRNA miR-122 as a therapeutic target for oligonucleotides and small molecules.
abstract: The most abundant microRNA (miRNA) in the liver, miR-122, is regulated by specific, liver-enriched transcription factors and is responsible for proper proliferation and differentiation of hepatocytes and for the regulation of lipid and cholesterol metabolisms. miR-122 is also involved in several hepatic disorders, as downregulation of miR-122 is often associated with hepatocellular carcinoma (HCC) and miR-122 is a required component for the replication and proliferation of the hepatitis C virus (HCV). Various probes have been developed to promote a better understanding of the involvement of miR-122 in liver diseases, including modified antisense agents and small molecule inhibitors. These agents, capable of specifically modifying miR-122 activity, provide excellent tools to investigate the function and regulation of miR-122 and offer potential new lead compounds for drug discovery. Especially small molecule modifiers can display numerous advantages over nucleotide analogs, as discussed in this review.
authors.affiliation: North Carolina State University, 2620 Yarbrough Drive, Raleigh, NC 27695, USA.
authors.forename: M
authors.initials: M
authors.lastname: Thomas
authors.forename: A
authors.initials: A
authors.lastname: Deiters
mesh_terms: D000818:Animals; D016174:Hepacivirus / Q000187:drug effects / Q000235:genetics / Q000502:physiology; D006801:Humans; D008107:Liver Diseases / Q000188:drug therapy / Q000235:genetics; D035683:MicroRNAs / Q000037:antagonists & inhibitors* / Q000235:genetics*; D058990:Molecular Targeted Therapy / Q000379:methods*; D016376:Oligonucleotides, Antisense / Q000235:genetics* / Q000627:therapeutic use*; D054852:Small Molecule Libraries / Q000494:pharmacology* / Q000627:therapeutic use
chemical_list: D035683:MicroRNAs; D016376:Oligonucleotides, Antisense; D054852:Small Molecule Libraries