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

作者：Thomas M; Deiters A^*

来源：Current Medicinal Chemistry, 2013, 20(29): 3629-3640.

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

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.

• 出版日期2013-9