title: miRNA therapeutics: a new class of drugs with potential therapeutic applications in the heart.
abstract: miRNAs are small non-coding RNAs (ncRNAs), which regulate gene expression. Here, the authors describe the contribution of miRNAs to cardiac biology and disease. They discuss various strategies for manipulating miRNA activity including antisense oligonucleotides (antimiRs, blockmiRs), mimics, miRNA sponges, Tough Decoys and miRNA mowers. They review developments in chemistries (e.g., locked nucleic acid) and modifications (sugar, 'ZEN', peptide nucleic acids) and miRNA delivery tools (viral vectors, liposomes, nanoparticles, pHLIP). They summarize potential miRNA therapeutic targets for heart disease based on preclinical studies. Finally, the authors review current progress of miRNA therapeutics in clinical development for HCV and cancer, and discuss challenges that will need to be overcome for similar therapies to enter the clinic for patients with cardiac disease.
authors.affiliation: Baker IDI Heart & Diabetes Institute, PO Box 6492, Melbourne, 3004, Australia.
authors.forename: Bianca C
authors.initials: BC
authors.lastname: Bernardo
authors.affiliation: Baker IDI Heart & Diabetes Institute, PO Box 6492, Melbourne, 3004, Australia.
authors.forename: Jenny Y Y
authors.initials: JY
authors.lastname: Ooi
authors.affiliation: Asbestos Diseases Research Institute, Sydney, 2139, Australia.|School of Biotechnology & Biomolecular Sciences, University of New South Wales, Sydney, 2052, Australia.
authors.forename: Ruby C Y
authors.initials: RC
authors.lastname: Lin
authors.affiliation: Baker IDI Heart & Diabetes Institute, PO Box 6492, Melbourne, 3004, Australia.|Departments of Medicine & Physiology, Monash University, Clayton, 3800, Australia.
authors.forename: Julie R
authors.initials: JR
authors.lastname: McMullen
mesh_terms: D000818:Animals; D002318:Cardiovascular Diseases / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D055808:Drug Discovery / Q000379:methods*; D005786:Gene Expression Regulation / Q000187:drug effects; D006321:Heart / Q000187:drug effects; D006801:Humans; D035683:MicroRNAs / Q000737:chemistry / Q000235:genetics / Q000494:pharmacology / Q000627:therapeutic use*; D009206:Myocardium / Q000473:pathology
chemical_list: D035683:MicroRNAs