摘要
Stability, specificity, and pharmacokinetic properties are some of the challenges facing RNAi therapeutics. In this review, the progresses in chemically modified siRNAs and siRNA conjugates are summarized. The proper modification of siRNA with nucleoside analogues, construction of siRNA conjugates, and reliable prediction of the property based on those strategies for a given siRNA sequence would certainly be an essential part of the solution to these challenges.
稳定性、特异性和药代动力学性质是发展RNA干扰治疗手段面临的三大主要问题。本文综述了目前干扰RNA化学修饰及其相关缀合物的研究进展,其核心工作是建立合适的siRNA的修饰/缀合策略,或者寻找方法能够预测修饰/缀合后siRNA的活性变化规律,以更好地解决该领域研究工作中的难点问题。
基金
National NaturalScience Foundation of China (Grant No.2093200 1,20832008)
National Basic Research Program of China(Grant No.2009ZX09503)