Right on target: The next class of efficient, safe, and specific RNAi triggers

Abstract

RNA interference (RNAi) is a powerful tool to control the expression of a target gene, yet RNAi triggers, typically small interfering (si) or short hairpin (sh) RNAs, are often prone to inducing adverse side effects that can limit their therapeutic index. By embedding Argonaute 2 (Ago2)-dependent shRNAs (agshRNAs) within a micro (mi)RNA backbone, and by thus enabling tissue-specific expression from an RNA polymerase (pol) II promoter, Alsing and colleagues have now taken a pivotal step at increasing the specificity and safety of therapeutic RNAi.