1 Department of Pathological Anatomy, Nantong University , Nantong, China .
Nucleic Acid Ther. 2014 Aug;24(4):302-12. doi: 10.1089/nat.2014.0480. Epub 2014 May 5.
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 siRNAs--in particular multi-target siRNAs--are reviewed.
近年来，RNA 干扰 (RNAi) 已被证明是一种针对各种未满足的医疗需求（如癌症）的治疗药物的新兴强大工具，特别是抗肿瘤药物的开发引起了极大的关注。最近在与癌症和眼部疾病相关的基于 RNAi 的治疗的临床试验中取得的成功验证了小干扰 RNA (siRNA) 构成了一种有前途的新一类治疗药物。目前，有大量基于多靶点的 siRNA 结构修饰方法可用于促进低副作用的 siRNA 介导的基因沉默。本文综述了基于 RNAi 的治疗方法的最新进展和针对 siRNA 的新型结构修饰（特别是多靶点 siRNA）。
