Biodistribution of phosphodiester and phosphorothioate siRNA

Biodistribution of phosphodiester and phosphorothioate siRNA
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DOI:
10.1016/j.bmcl.2003.12.074
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发表时间:
2004-03-08
影响因子:
2.7
通讯作者:
Corey, DR
Corey, DR
中科院分区:
医学4区
文献类型:
--
作者:
Braasch, DA;Paroo, Z;Corey, DR

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短干扰rna (sirna)是分析哺乳动物细胞培养中蛋白质功能的重要工具。这一成功导致了对体内和治疗应用的高期望。然而,siRNA的药代动力学性质尚不清楚。在这里,我们报告了一个磷酸二酯(PO) siRNA双链的生物分布,并检查了磷硫(PS)键的影响。我们的研究结果表明,siRNA的生物分布与单链反义寡核苷酸相似,并为siRNA在体内的使用提供了见解。(C) 2003 Elsevier Ltd.版权所有。
Short interfering RNAs (siRNAs) are valuable tools for analyzing protein function in mammalian cell culture. This success has led to high expectations for in vivo and therapeutic applications. However, the pharmacokinetic properties of siRNA are not known. Here we report the biodistribution of a phosphodiester (PO) siRNA duplex and examine the effect of phosphorothioate (PS) linkages. Our findings indicate that biodistribution of siRNA is similar to that for single-stranded antisense oligonucleotides and offer insights for use of siRNA in vivo. (C) 2003 Elsevier Ltd. All rights reserved.