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小干扰RNA导入哺乳动物细胞的研究

On the delivery of small interfering RNAs into mammalian cells.

作者信息

Sioud Mouldy

机构信息

The Norwegian Radium Hospital, Department of Immunology, Molecular Medicine Group, Montebello, Oslo.

出版信息

Expert Opin Drug Deliv. 2005 Jul;2(4):639-51. doi: 10.1517/17425247.2.4.639.

Abstract

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.

摘要

RNA干扰正成为分析基因功能和验证药物靶点的首选技术。在这个过程中,序列特异性基因抑制由小RNA双链体引发,即所谓的小干扰RNA(siRNAs)。外源性递送的siRNAs或内源性表达的发夹状siRNAs能够在体外和动物模型中导致特定靶mRNA的降解,这一点已经得到证实。然而,将siRNAs开发为人类治疗药物的关键挑战很大程度上取决于合适递送载体的开发以及siRNA特异性的提高。本综述重点介绍了siRNA递送方面的最新进展以及与免疫刺激相关的挑战性问题。

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