MicroRNA miR-122 as a Therapeutic Target for Oligonucleotides and Small Molecules

Author(s): M. Thomas, A. Deiters

Journal Name: Current Medicinal Chemistry

Volume 20 , Issue 29 , 2013

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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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Article Details

Year: 2013
Published on: 11 August, 2013
Page: [3629 - 3640]
Pages: 12
DOI: 10.2174/0929867311320290009
Price: $65

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