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基于载体的小干扰RNA递送:体外和体内面临的挑战。

Vector-based delivery of siRNAs: in vitro and in vivo challenges.

作者信息

Walchli Sebastien, Sioud Mouldy

机构信息

Institute for Cancer Research, Department of Immunology, Molecular Medicine Group, Rikshospitalet-Radiumhospitalet Medical Centre, Montebello 0310 Oslo, Norway.

出版信息

Front Biosci. 2008 May 1;13:3488-93. doi: 10.2741/2943.

DOI:10.2741/2943
PMID:18508450
Abstract

RNA interference (RNAi) induced by small interfering RNAs (siRNAs) has recently become a powerful tool to knock-down gene expression in a sequence-specific manner. In addition to chemically made siRNAs, stable expression of siRNA in the form of short hairpin RNAs (shRNAs) expressed from an RNA polymerase III (pol III) promoter is now widely used approach for the application of RNAi in mammalian cells. However, long-term suppression using constitutive promoters can be problematic and emerging evidence indicates that siRNAs can cause several side effects in human cells. Here we review the recent advances in developing controllable expression vectors in order to accelerate the therapeutic applications of RNAi.

摘要

由小干扰RNA(siRNA)诱导的RNA干扰(RNAi)最近已成为一种以序列特异性方式敲低基因表达的强大工具。除了化学合成的siRNA外,以RNA聚合酶III(pol III)启动子表达的短发夹RNA(shRNA)形式稳定表达siRNA,现在是RNAi在哺乳动物细胞中应用的广泛使用的方法。然而,使用组成型启动子进行长期抑制可能存在问题,并且新出现的证据表明siRNA可在人类细胞中引起多种副作用。在这里,我们综述了在开发可控表达载体方面的最新进展,以加速RNAi的治疗应用。

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Vector-based delivery of siRNAs: in vitro and in vivo challenges.基于载体的小干扰RNA递送:体外和体内面临的挑战。
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Indoleamine-2,3-dioxygenase, an immunosuppressive enzyme that inhibits natural killer cell function, as a useful target for ovarian cancer therapy.色氨酸 2,3-双加氧酶,一种抑制自然杀伤细胞功能的免疫抑制酶,是卵巢癌治疗的一个有用靶点。
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