无环苏氨酸醇核酸修饰RNA,用于RNA干扰

A. Alagia, M. Terrazas, R. Eritja
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引用次数: 0

摘要

尽管合成小干扰RNA (siRNA)已被广泛用于下调任何蛋白质编码mRNA,但仍有几个关键问题尚未解决。无环苏氨酸醇核酸(aTNA)被放置在siRNA的特定位置,是减少寡核苷酸对核酸酶易损性的有用修饰。此外,可以利用它来避免几种限制基于rnai的药物的生物安全性的脱靶效应。
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RNA modified with acyclic threoninol nucleic acids for RNA interference
Although synthetic small interfering RNA (siRNA) has been extensively used to downregulate any protein-coding mRNA, several key issues still remain unsolved. The acyclic threoninol nucleic acid (aTNA), placed at certain siRNA positions, is a useful modification to reduce the oligonucleotides vulnerability towards nucleases. In addition, it can be exploited to avoid several OFF-target effects that limit the biological safety of the RNAi-based agents.
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