Chemical modification: the key to clinical application of RNA interference?

被引：231

作者：

Corey, David R. ^{[1
]}

机构：

[1] Univ Texas SW Med Ctr Dallas, Dept Pharmacol, Dallas, TX 75390 USA

[2] Univ Texas SW Med Ctr Dallas, Dept Biochem, Dallas, TX 75390 USA

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2007年 / 117卷 / 12期

关键词：

D O I：

10.1172/JCI33483

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

RNA interference provides a potent and specific method for controlling gene expression in human cells. To translate this potential into a broad new family of therapeutics, it is necessary to optimize the efficacy of the RNA-based drugs. As discussed in this Review, it might be possible to achieve this optimization using chemical modifications that improve their in vivo stability, cellular delivery, biodistribution, pharmacokinetics, potency, and specificity.

引用

页码：3615 / 3622

页数：8

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