Therapeutic siRNAs

被引:98
作者
Sioud, M [1 ]
机构
[1] Norwegian Radium Hosp, Dept Immunol, Mol Med Grp, N-310 Oslo, Norway
关键词
D O I
10.1016/j.tips.2003.11.006
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
The ability of small-interfering RNAs (siRNAs) to silence gene expression in somatic mammalian cells has provided researchers with a novel tool to block the expression of disease-causing genes, provided that their mRNA sequences are known. siRNAs can be delivered to cells either exogenously as synthetic agents or endogenously as gene-encoding siRNAs. Recent studies demonstrate the general application of siRNAs to silence gene expression in a range of cell types and in whole mammals. Beyond their value for dissecting gene functions and target validation, siRNAs also hold great potential as gene-specific therapeutic agents.
引用
收藏
页码:22 / 28
页数:7
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