title: miRNA Targeting Drugs: The Next Blockbusters?
abstract: Only 20 years after the discovery of small non-coding, single-stranded ribonucleic acids, so-called microRNAs (miRNAs), as post-transcriptional gene regulators, the first miRNA-targeting drug Miravirsen for the treatment of hepatitis C has been successfully tested in clinical Phase II trials. Addressing miRNAs as drug targets may enable the cure, or at least the treatment of diseases, which presently seems impossible. However, due to miRNAs' chemical structure, generation of potential drug molecules with necessary pharmacokinetic properties is still challenging and requires a re-thinking of the drug discovery process. Therefore, this chapter highlights the potential of miRNAs as drug targets, discusses the challenges, and tries to give a complete overview of recent strategies in miRNA drug discovery.
authors.affiliation: BEROCEUTICA GmbH, c/o Universität Potsdam, August-Bebel-Strasse 89, 14482, Potsdam, Germany. marco.schmidt@beroceutica.com.
authors.forename: Marco F
authors.initials: MF
authors.lastname: Schmidt
mesh_terms: D016503:Drug Delivery Systems / Q000379:methods*; D055808:Drug Discovery; D005786:Gene Expression Regulation; D016174:Hepacivirus / Q000187:drug effects / Q000472:pathogenicity; D006526:Hepatitis C / Q000235:genetics / Q000628:therapy*; D006801:Humans; D035683:MicroRNAs / Q000235:genetics / Q000627:therapeutic use*; D009841:Oligonucleotides / Q000627:therapeutic use
chemical_list: D035683:MicroRNAs; D009841:Oligonucleotides; C581159:miravirsen
keywords: Antisense agents; Argonaute 2 protein; Drug discovery; Small-molecule miRNA modulators; miRNA; microRNA-induced silencing complex