Recent development of RNAi in drug target discovery and validation

This article reviews the recent development in two areas of RNAi technology that could potentially shorten the otherwise long process of drug development. First, the combinatorial gene silencing built on the RNAi library, particularly the coming of age of ‘RNAi gene’ libraries, by overcoming numerous technical hurdles and also increasing understanding of miRNA biogenesis, is gradually establishing more effective approaches to identifying drug targets. Second, regulated RNAi gene expression provides tools for in vivo target validation for both on-target efficacy and potential on-target toxicity.

Section editors:Li-he Zhang – School of Pharmaceutical Science, Peking University, Beijing, ChinaKaixian Chen – Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai, China