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SIRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAI, AND STABILIZED FORMS THEREOF

机译:用于减少RNAi中脱靶表型效应的SIRNA序列独立修饰格式及其稳定的形式

摘要

Modification formats having modified nucleotides are provided for siRNA. Short interfering RNA having modification formats and modified nucleotides provided herein reduce off-target effects in RNA interference of endogenous genes. Further modification formatted siRNAs are demonstrated to be stabilized to nuclease-rich environments. Unexpectedly, increasing or maintaining strand bias, while necessary to maintain potency for endogenous RNA interference, is not sufficient for reducing off-target effects in cell biology assays.
机译:为siRNA提供了具有修饰的核苷酸的修饰形式。本文提供的具有修饰形式和修饰核苷酸的短干扰RNA减少了内源基因RNA干扰中的脱靶效应。进一步的修饰格式化的siRNA被证明可以稳定在富含核酸酶的环境中。出乎意料的是,增加或维持链偏向性,尽管对于维持内源RNA干扰的效力是必要的,但不足以减少细胞生物学测定中的脱靶效应。

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