title: Discovering the first microRNA-targeted drug.
abstract: MicroRNAs (miRNAs) are important post-transcriptional regulators of nearly every biological process in the cell and play key roles in the pathogenesis of human disease. As a result, there are many drug discovery programs that focus on developing miRNA-based therapeutics. The most advanced of these programs targets the liver-expressed miRNA-122 using the locked nucleic acid (LNA)-modified antisense oligonucleotide miravirsen. Here, we describe the discovery of miravirsen, which is currently in phase 2 clinical trials for treatment of hepatitis C virus (HCV) infection.
authors.affiliation: Department of Biology, The Bioinformatics Centre, University of Copenhagen, DK-2200 Copenhagen, Denmark.
authors.forename: Morten
authors.initials: M
authors.lastname: Lindow
authors.forename: Sakari
authors.initials: S
authors.lastname: Kauppinen
mesh_terms: D005786:Gene Expression Regulation; D016174:Hepacivirus / Q000235:genetics; D006526:Hepatitis C / Q000235:genetics / Q000628:therapy*; D006801:Humans; D008099:Liver / Q000378:metabolism*; D035683:MicroRNAs / Q000235:genetics*; D058990:Molecular Targeted Therapy; D009841:Oligonucleotides / Q000008:administration & dosage; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use*
chemical_list: D035683:MicroRNAs; D009841:Oligonucleotides; D016376:Oligonucleotides, Antisense; C477371:locked nucleic acid