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 up-regulation of miRNAs is achieved through administration of synthetic miRNAs or administration of miRNA expressing vectors. The down-regulation 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 miRNAs-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.
Keywords: Antagomir; Cancer; Fibrosis; Hepatocellular carcinoma; Inhibitor; Mimic; Off-target effect; OncomiRNAs; Pathobiology; SBMA; Spinal and bulbar muscular atrophy; TSmiRNAs; Therapy; Tumor suppressor miRNAs; miRNA; microRNA.