首页> 外国专利> Nucleic acid that induces RNA interference modified to prevent off-target, composition for suppressing gene expression, kit for suppressing gene expression, method for suppressing expression of intracellular target gene, method for suppressing off-target effect

Nucleic acid that induces RNA interference modified to prevent off-target, composition for suppressing gene expression, kit for suppressing gene expression, method for suppressing expression of intracellular target gene, method for suppressing off-target effect

机译:修饰为防止脱靶而诱导RNA干扰的核酸,用于抑制基因表达的组合物,用于抑制基因表达的试剂盒,用于抑制细胞内靶基因表达的方法,用于抑制脱靶效应的方法

摘要

The present invention relates to an RNA interference-inducing nucleic acid and the use thereof, and more particularly to an RNA interference-inducing nucleic acid comprising at least one single strand of double strands, the at least one single strand comprising a modification substituted to a spacer, which is unable to form a base pair, in the 5' end or the 3' end region. The RNA interference-inducing nucleic acid of the present invention is a novel modified form of nucleotide provided to prevent off-target effects, offering a method to selectively repress target gene expression. In addition, according to the present invention, the RNA interference-inducing nucleic acid provides novel modified forms with target selectivity and specificity as a method to block the off-target effects while silencing the target gene expression, whereas the usage of conventional RNA interference-inducing nucleic acids cause inaccuracy and adverse effects through off-targets, thereby the RNA interference-inducing nucleic acid was offered to solve the problem, wherein it will be widely used as a method for repressing gene expression in research and for gene therapy without concerning the off-target effects.
机译:本发明涉及一种诱导RNA干扰的核酸及其用途,更具体地,涉及一种包含至少一条双链单链的RNA干扰诱导核酸,该至少一条单链包含被α-α取代的修饰。在5'末端或3'末端区域中不能形成碱基对的间隔基。本发明的诱导RNA干扰的核酸是核苷酸的新型修饰形式,其被提供以防止脱靶效应,从而提供了选择性抑制靶基因表达的方法。此外,根据本发明,诱导RNA干扰的核酸可提供具有靶标选择性和特异性的新型修饰形式,作为一种在沉默靶基因表达的同时阻断脱靶效应的方法,而常规RNA干扰的用途是诱导核酸通过脱靶引起不准确和不利影响,从而提供了解决RNA干扰的核酸,从而解决了这一问题,它将被广泛用作研究和基因治疗中抑制基因表达的方法而无需考虑脱靶效应。

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