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化学修饰的干扰RNA及其缀合物

陈玥, 王晓峰, 黄野, 张礼和, 杨振军*   

  1. 北京大学医学部 天然药物及仿生药物国家重点实验室, 北京 100191
  • 收稿日期:2011-11-11 修回日期:2011-12-10 出版日期:2012-01-01 发布日期:2012-01-01
  • 通讯作者: 杨振军*

Chemically modified siRNAs and their conjugates

Yue Chen, Xiao-Feng Wang, Ye Huang, Li-He Zhang, Zhen-Jun Yang*   

  1. State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Beijing 100191, China
  • Received:2011-11-11 Revised:2011-12-10 Online:2012-01-01 Published:2012-01-01
  • Contact: Zhen-Jun Yang*

摘要: 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。

关键词: siRNA, 化学修饰siRNA, 生物缀合siRNA

Abstract: 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.

Key words: siRNA, Chemical modified siRNA, Conjugated siRNA

中图分类号: 

Supporting:

Foundation items: National Natural Science Foundation of China (Grant No. 20932001, 20832008), and National Basic Research Program of China (Grant No. 2009ZX09503).
*Corresponding author. Tel.: 86-10-82802503