title: MicroRNA-targeting therapeutics for hepatitis C.
abstract: MiR-122 is a liver-specific microRNA (miRNA) that plays a pivotal role in regulating hepatic functions such as lipid metabolism and stress response. The observation that hepatitis C virus (HCV) could only replicate in miR-122-positive hepatocytes led to the discovery that miR-122 is essential for HCV replication, and miR-122 is now one of the crucial host factors for anti-HCV therapy. Currently, the most advanced miR-122 targeting therapy is SPC3649 (miravirsen), a locked nucleic acid-modified oligonucleotide antagonizing miR-122. This review serves to provide information on the discovery and development of SPC3649, the first miRNA-targeted drug to enter human clinical trials, and introduce other miR-122-targeting therapeutics being developed for hepatitis C.
authors.affiliation: College of Pharmacy, Chung-Ang University, 84 Heukseokro, Dongjakgu, Seoul, 156-756, Korea.
authors.forename: Jihae
authors.initials: J
authors.lastname: Baek
authors.forename: Soowon
authors.initials: S
authors.lastname: Kang
authors.forename: Hyeyoung
authors.initials: H
authors.lastname: Min
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic / Q000379:methods; D016503:Drug Delivery Systems / Q000639:trends*; D015507:Drugs, Investigational / Q000008:administration & dosage; D006526:Hepatitis C / Q000188:drug therapy* / Q000235:genetics; D006801:Humans; D035683:MicroRNAs / Q000037:antagonists & inhibitors* / Q000502:physiology; D009841:Oligonucleotides; D054735:Phosphorothioate Oligonucleotides / Q000008:administration & dosage*
chemical_list: D015507:Drugs, Investigational; C531907:MIRN122 microRNA, human; D035683:MicroRNAs; D009841:Oligonucleotides; D054735:Phosphorothioate Oligonucleotides; C581159:miravirsen