miRNA-Based Therapeutic Strategies

Abstract

Micro-RNAs (miRNAs) are short, non-coding RNA species, thought to act primarily through downregulation of target mRNA species with subsequent decrease in encoded proteins. Recent studies revealed that miRNAs play pivotal roles in physiology and disease, and therapeutic targeting has started being investigated. Generally, the upregulation of miRNAs is achieved through administration of synthetic miRNAs or administration of miRNA expressing vectors. The downregulation of miRNAs is achieved through administration of anti-sense nucleotides, often chemically modified to ensure stability and specificity. There are multiple potential limitations associated with the development and testing of miRNA-based therapeutics. These issues include, but are not limited to, off-target effect, avoidance from internal nucleases, and toxicity for miRNA therapy. In this review, we will discuss recent advances in miRNA based therapeutic strategies.