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Future Med Chem. 2015;7(13):1771-92. doi: 10.4155/fmc.15.107. Epub 2015 Sep 24.

miRNA therapeutics: a new class of drugs with potential therapeutic applications in the heart.

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Baker IDI Heart & Diabetes Institute, PO Box 6492, Melbourne, 3004, Australia.
Asbestos Diseases Research Institute, Sydney, 2139, Australia.
School of Biotechnology & Biomolecular Sciences, University of New South Wales, Sydney, 2052, Australia.
Departments of Medicine & Physiology, Monash University, Clayton, 3800, Australia.


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.

[Indexed for MEDLINE]

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