MicroRNA: an Emerging Therapeutic Target and Intervention Tool

TLDR

MicroRNAs are short non‑coding RNAs that regulate over one third of human mRNAs, with more than 600 identified, and their dysregulation is linked to cancers, neurodegenerative, cardiovascular, and viral diseases, making them attractive therapeutic targets via decoys, sponges, expression vectors, mimetics, and precursor scaffolds. This review examines recent advances in miRNA‑based therapeutics. It discusses how synthetic decoys, sponges, expression vectors, mimetics, and precursor scaffolds are employed to modulate gene expression.

Abstract

MicroRNAs (miRNAs) are a class of short non-coding RNAs with posttranscriptional regulatory functions. To date, more than 600 human miRNAs have been experimentally identified, and estimated to regulate more than one third of cellular messenger RNAs. Accumulating evidence has linked the dysregulated expression patterns of miRNAs to a variety of diseases, such as cancer, neurodegenerative diseases, cardiovascular diseases and viral infections. MiRNAs provide its particular layer of network for gene regulation, thus possessing the great potential both as a novel class of therapeutic targets and as a powerful intervention tool. In this regard, synthetic RNAs that contain the binding sites of miRNA have been shown to work as a "decoy" or "miRNA sponge" to inhibit the function of specific miRNAs. On the other hand, miRNA expression vectors have been used to restore or overexpress specific miRNAs to achieve a long-term effect. Further, double-stranded miRNA mimetics for transient replacement have been experimentally validated. Endogenous precursor miRNAs have also been used as scaffolds for the induction of RNA interference. This article reviews the recent progress on this emerging technology as a powerful tool for gene regulation studies and particularly as a rationale strategy for design of therapeutics.