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.
Keywords: miRNA-122, liver disorders, miRNA therapeutics, small molecules, antisense oligonucleotides, antagomirs, hepatitis
C virus, hepatocellular carcinoma, lipid and cholesterol metabolism, regulation of miRNA function.
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