RNA Interference in Cancer Therapy

Abstract

Current research in cancer therapeutics is leading the way in exploiting the growing amounts of genetic and molecular information on human oncobiology to design new anticancer agents to target specific tumor-related signaling pathways, and to treat patients according to their clinical characteristics and molecular profile. In this context, RNA interference (RNAi), a ubiquitous cellular pathway of post-transcriptional gene regulation, provides an intriguing tool for an innovative rational cancer drug design. Among the endogenous mediators of RNAi, microRNAs (miRNAs) represent the most important class of small RNAs whose global dysregulation is a typical feature of human tumors. Harnessing of the RNAi machinery by using small, synthetic RNAs that target or mimic endogenous miRNAs offers the opportunity to reach virtually any gene and pathway relevant to tumor maintenance. However, along with assessments of the safety profile and effectiveness, strategies for assuring the specificity of these drugs, in terms of their effective delivery to cancer cells, must be devised before RNAi formulations can be made into ideal personalized therapeutics for the clinic. Keywords: cancer; RNA interference; RISC (RNA-induced silencing complex); microRNAs (miRNAs; miRs); siRNAs; antimiRs; personalized medicine; RNAi-based therapy