Short Interfering RNA (siRNA) based Medicines and the Future of RNAi Therapy: A Mini Review

Abstract

After twenty years of short interfering RNA (siRNA) research, RNA interference (RNAi)-based medicines are emerging as a novel class of drugs since the recent approval of Onpattro® and Givlaari® by the Food and Drug Administration (FDA). RNAi is a post-transcriptional gene regulatory process in which one of its molecular effectors is a silencing inducer: the siRNA. Since the description of this pathway, several research groups are searching for therapeutic applications of siRNA against diseases with overexpressed genes. Many challenges came across and strategies to improve siRNA stability and delivery were developed. Nevertheless, higher efficacy, safety and successful delivery are still some of the challenges to be overcome. In this mini review, we are presenting some of the biological barriers for effective siRNA delivery as well as an update of the current progress in RNAi-based medicines in phase III clinical trials.