title: miRNA-based therapies: strategies and delivery platforms for oligonucleotide and non-oligonucleotide agents.
abstract: The discovery of miRNAs as important regulatory agents for gene expression has expanded the therapeutic opportunities for oligonucleotides. In contrast to siRNA, miRNA-targeted therapy is able to influence not only a single gene, but entire cellular pathways or processes. It is possible to supplement downregulated or non-functional miRNAs by synthetic oligonucleotides, as well as alleviating effects caused by overexpression of malignant miRNAs through artificial antagonists, either oligonucleotides or small molecules. Chemical oligonucleotide modifications together with an efficient delivery system seem to be mandatory for successful therapeutic application. While miRNA-based therapy benefits from the decades of research spent on other therapeutic oligonucleotides, there are some specific challenges associated with miRNA therapy, mainly caused by the short target sequence. The current status and recent progress of miRNA-targeted therapeutics is described and future challenges and potential applications in treatment of cancer and viral infections are discussed.
authors.affiliation: University of Vienna, Department of Pharmaceutical Chemistry, Althanstraße 14, 1090 Vienna, Austria.
authors.forename: Volker
authors.initials: V
authors.lastname: Baumann
authors.forename: Johannes
authors.initials: J
authors.lastname: Winkler
mesh_terms: D004337:Drug Carriers / Q000737:chemistry*; D006801:Humans; D008081:Liposomes / Q000737:chemistry; D035683:MicroRNAs / Q000037:antagonists & inhibitors / Q000378:metabolism*; D009369:Neoplasms / Q000188:drug therapy / Q000235:genetics; D009841:Oligonucleotides / Q000737:chemistry* / Q000627:therapeutic use; D011108:Polymers / Q000737:chemistry; D012330:RNA, Double-Stranded / Q000737:chemistry / Q000627:therapeutic use; D034741:RNA, Small Interfering / Q000737:chemistry / Q000627:therapeutic use; D014777:Virus Diseases / Q000188:drug therapy / Q000235:genetics
chemical_list: D004337:Drug Carriers; D008081:Liposomes; D035683:MicroRNAs; D009841:Oligonucleotides; D011108:Polymers; D012330:RNA, Double-Stranded; D034741:RNA, Small Interfering; C477371:locked nucleic acid