Development of RNA Interference-Based Therapeutics and Application of Multi-Target Small Interfering RNAs

作者:Li Tiejun; Wu Meihua; Zhu York Yuanyuan; Chen Jianxin; Chen Li*
来源:Nucleic Acid Therapeutics, 2014, 24(4): 302-312.
DOI:10.1089/nat.2014.0480

摘要

RNA interference (RNAi) has been proven in recent years to be a newly advanced and powerful tool for development of therapeutic agents toward various unmet medical needs such as cancer, in particular, a great attention has been paid to the development of antineoplastic agents. Recent success in clinical trials related to RNAi-based therapeutics on cancer and ocular disease has validated that small interfering RNAs (siRNAs) constitute a new promising class of therapeutics. Currently, a great wealth of multi-target based siRNA structural modifications is available for promoting siRNA-mediated gene silencing with low side effects. Here, the latest developments in RNAi-based therapeutics and novel structural modifications described for siRNA-sin particular multi-target siRNAs-are reviewed.