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A novel measurement of allele discrimination for assessment of allele-specific silencing by RNA interference

机译:一种通过RNA干扰评估等位基因特异性沉默的等位基因歧视的新方法

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摘要

Allele-specific silencing by RNA interference (ASP-RNAi) is an atypical RNAi that is capable of discriminating target alleles from non-target alleles, and may be therapeutically useful for specific inhibition of disease-causing alleles without affecting their corresponding normal alleles. However, it is difficult to design and select small interfering RNA (siRNAs) that confer ASP-RNAi. A major problem is that there are few appropriate measures in determining optimal allele-specific siRNAs. Here we show two novel formulas for calculating a new measure of allele-discrimination, named "ASP-score''. The formulas and ASP-score allow for an unbiased determination of optimal siRNAs, and may contribute to characterizing such allele-specific siRNAs.
机译:RNA干扰引起的等位基因特异性沉默(ASP-RNAi)是一种非典型RNAi,能够将目标等位基因与非目标等位基因区分开,并且在治疗致病性等位基因而不影响其相应正常等位基因方面可能具有治疗作用。但是,很难设计和选择赋予ASP-RNAi的小干扰RNA(siRNA)。一个主要问题是,在确定最佳等位基因特异性siRNA时几乎没有适当的措施。在这里,我们展示了两个新颖的公式,用于计算一种新的等位基因差异测量方法,称为“ ASP评分”,该公式和ASP评分可无偏确定最佳siRNA,并可能有助于表征此类等位基因特异性siRNA。

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