siRNA的化学修饰。

Elena L Chernolovskaya, Marina A Zenkova
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引用次数: 0

摘要

sirna在分子生物学和实验药理学中广泛应用于基因表达调控。siRNA分子是目前发现的最有效和特异性的基因表达抑制剂,在纳摩尔和亚纳摩尔浓度下起作用。与核酸酶稳定性、选择有效序列、减少脱靶效应以及实现靶细胞和组织的高效递送相关的挑战,极大地限制了siRNA在生物医学上的应用。然而,化学修饰是提高siRNA性能的有力工具。本文综述了最近关于化学修饰用于开发基因沉默的有效效应器的数据。讨论了热稳定性与效价、核酸酶抗性与沉默效应持续时间的相关性,以及与化学修饰sirna的关系。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Chemical modification of siRNA.

siRNAs are widely used for the control of gene expression in molecular biology and experimental pharmacology. siRNA molecules are the most efficient and specific inhibitors of gene expression to have been discovered, acting at nanomolar and subnanomolar concentrations. Challenges associated with nuclease stability, selecting effective sequences, reducing off-target effects and achieving efficient delivery to target cells and tissues have significantly limited the biomedical applications of siRNA. However, chemical modification is a powerful tool for improving the properties of siRNA. This review examines recent data regarding chemical modifications used to develop potent effectors of gene silencing. The correlation between thermal stability and potency, and between nuclease resistance and duration of the silencing effect, are discussed with relation to chemically modified siRNAs.

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来源期刊
Current Opinion in Molecular Therapeutics
Current Opinion in Molecular Therapeutics 医学-生物工程与应用微生物
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