RNA interference and chemically modified small interfering RNAs

被引:296
作者
Manoharan, M [1 ]
机构
[1] Alnylam Pharmaceut Inc, Cambridge, MA 02142 USA
关键词
D O I
10.1016/j.cbpa.2004.10.007
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
RNA interference (RNAi) is a powerful biological process for specific silencing of gene expression in diversified eukaryotic cells and has tremendous potential for functional genomics, drug discovery through in vivo target validation, and development of novel gene-specific medicine. The future success of this technology relies on identifying appropriate chemical modifications to improve stability, potency and in vivo cellular delivery. The present review summarizes the role of the chemist's toolbox in this emerging technology.
引用
收藏
页码:570 / 579
页数:10
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