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Nat Rev Drug Discov. 2014 Aug;13(8):622-38. doi: 10.1038/nrd4359. Epub 2014 Jul 11.

Therapeutic targeting of microRNAs: current status and future challenges.

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Drug Safety, Research &Development, Pfizer, 1 Burtt Road, Andover, Massachusetts 01845, USA.
1] Program for RNA Biology, Sanford-Burnham Medical Research Institute, 10901 North Torrey Pines Road, La Jolla, California 92037, USA. [2] Department of Pediatrics, University of California San Diego School of Medicine, 9500 Gilman Drive MC 0762, La Jolla, California 92093, USA.


MicroRNAs (miRNAs) are evolutionarily conserved small non-coding RNAs that have crucial roles in regulating gene expression. Increasing evidence supports a role for miRNAs in many human diseases, including cancer and autoimmune disorders. The function of miRNAs can be efficiently and specifically inhibited by chemically modified antisense oligonucleotides, supporting their potential as targets for the development of novel therapies for several diseases. In this Review we summarize our current knowledge of the design and performance of chemically modified miRNA-targeting antisense oligonucleotides, discuss various in vivo delivery strategies and analyse ongoing challenges to ensure the specificity and efficacy of therapeutic oligonucleotides in vivo. Finally, we review current progress on the clinical development of miRNA-targeting therapeutics.

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