North Carolina State University, 2620 Yarbrough Drive, Raleigh, NC 27695, USA.
Curr Med Chem. 2013;20(29):3629-40. doi: 10.2174/0929867311320290009.
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.
肝脏中含量最丰富的 microRNA（miRNA）是 miR-122，它受特定的肝脏丰富转录因子调控，负责肝细胞的正常增殖和分化，以及脂质和胆固醇代谢的调节。miR-122 还与几种肝脏疾病有关，因为 miR-122 的下调通常与肝细胞癌（HCC）有关，miR-122 是丙型肝炎病毒（HCV）复制和增殖所必需的组成部分。已经开发了各种探针来促进更好地理解 miR-122 在肝脏疾病中的作用，包括修饰的反义剂和小分子抑制剂。这些能够特异性修饰 miR-122 活性的试剂为研究 miR-122 的功能和调节提供了极好的工具，并为药物发现提供了潜在的新先导化合物。正如本文所讨论的，小分子调节剂相对于核苷酸类似物具有许多优势。
