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RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs

by Sarah Bajan 1,2,* and Gyorgy Hutvagner 3,*
Faculty of Science, University of Technology Sydney, Sydney, NSW 2000, Australia
Health and Sport Science, University of Sunshine Coast, Sunshine Coast, QLD 4556, Australia
School of Biomedical Engineering Faculty of Engineering and Information Technology, University of Technology Sydney, Sydney, NSW 2000, Australia
Authors to whom correspondence should be addressed.
Cells 2020, 9(1), 137; (registering DOI)
Received: 28 November 2019 / Revised: 23 December 2019 / Accepted: 30 December 2019 / Published: 7 January 2020
(This article belongs to the Special Issue microRNA as Therapeutic Target)
The first therapeutic nucleic acid, a DNA oligonucleotide, was approved for clinical use in 1998. Twenty years later, in 2018, the first therapeutic RNA-based oligonucleotide was United States Food and Drug Administration (FDA) approved. This promises to be a rapidly expanding market, as many emerging biopharmaceutical companies are developing RNA interference (RNAi)-based, and RNA-based antisense oligonucleotide therapies. However, miRNA therapeutics are noticeably absent. miRNAs are regulatory RNAs that regulate gene expression. In disease states, the expression of many miRNAs is measurably altered. The potential of miRNAs as therapies and therapeutic targets has long been discussed and in the context of a wide variety of infections and diseases. Despite the great number of studies identifying miRNAs as potential therapeutic targets, only a handful of miRNA-targeting drugs (mimics or inhibitors) have entered clinical trials. In this review, we will discuss whether the investment in finding potential miRNA therapeutic targets has yielded feasible and practicable results, the benefits and obstacles of miRNAs as therapeutic targets, and the potential future of the field. View Full-Text
Keywords: miRNA; RNAi; drug delivery; Rna therapeutics; antisense RNA miRNA; RNAi; drug delivery; Rna therapeutics; antisense RNA
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Bajan, S.; Hutvagner, G. RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs. Cells 2020, 9, 137.

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