On the delivery of small interfering RNAs into mammalian cells.
On the delivery of small interfering RNAs into mammalian cells.
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DOI：
10.1517/17425247.2.4.639
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发表时间：
2005-07-01
影响因子：
6.6
通讯作者：
Sioud, Mouldy
中科院分区：
文献类型：
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作者：
Sioud, Mouldy
RNA干扰正成为分析基因功能和药物靶点验证的首选技术。在这个过程中，序列特异性基因抑制是由小RNA双链体启动的，称为小干扰RNA（siRNA）。已经证明了外源递送的siRNA或内源表达的发夹siRNA可以在体外和动物模型中引起特异性靶mRNA的破坏的可能性。然而，开发siRNA作为人类治疗剂的关键挑战在很大程度上取决于开发合适的递送剂和改进的siRNA特异性。这篇综述强调了siRNA递送的最新进展，以及与免疫刺激相关的挑战性问题。
RNA interference is becoming the technique of choice for analysing gene function and drug target validation. In this process, sequence-specific gene inhibition is initiated by small RNA duplexes, known as small interfering RNAs (siRNAs). The possibility that exogenously delivered siRNAs or endogenously expressed hairpin siRNAs can cause the destruction of specific target mRNA in vitro and in animal models has been demonstrated. However, the key challenges for the development of siRNAs as human therapeutics is largely dependent on the development of suitable delivery agents and improved siRNA specificity. This review highlights recent advances in siRNA delivery, as well as challenging problems related to immune stimulation.