A narrative review of microRNA therapeutics: understanding the future of microRNA research

Abstract

Objective: In this review, we will discuss miRNA therapeutics in clinical trials and the current challenges of miRNA therapeutics. Background: MicroRNAs (miRNAs) play crucial roles in the development, proliferation, differentiation, survival and cell metabolism. Many miRNAs are frequently changed in the pathological processes, including cancers. The powerful regulation of miRNAs on the transcriptome enables basic and translational studies of miRNAs on the clinical management of cancer. Methods: The information used in this narrative review was collected from published literatures and ongoing/completed clinical trials (clinicalTrials.gov). Conclusions: One advantage of miRNA therapeutic is that one single miRNA can target multiple genes, which makes miRNA a powerful regulator for transcriptome. A great number of studies indicate that miRNAs control the development and progression of cancers. Despite the great success of miRNA therapeutics in pre-clinical studies, a little number of clinical trials are conducted in the present. Currently, the major challenges of miRNA therapeutics are the delivery of miRNA memetic/inhibitor molecules and the immune effects induced by the miRNA drugs. Chemical modification, conjugation of various moieties and package into the nanoparticles can abrogate the adverse effects (AEs) induced miRNA drugs and increase the delivery efficiency. With these approaches, it is promising that miRNA therapeutics may be successfully used in primary cancer therapeutics in the near future.