发明名称 INHIBITOR OF EXPRESSION OF DOMINANTLY MUTATED GENE
摘要 It is intended to develop an RNAi molecule that can selectively and effectively suppress only the expression of a particular dominant mutant gene, while permitting the expression of the wild-type gene or a desired mutant gene, and to provide a design method thereof and an agent for suppressing the expression of a dominant mutant gene, comprising the RNAi molecule as an active ingredient. The present invention provides an agent for suppressing the expression of a dominant mutant gene, comprising an RNAi molecule against the dominant mutant gene generating a point of discontinuity on a transcript, the RNAi molecule having a predetermined length set as a base length from a base 3' flanking the point of discontinuity on a sense strand region to the 3' end of the sense strand region.
申请公布号 EP2623600(A4) 申请公布日期 2014.11.26
申请号 EP20110829186 申请日期 2011.09.28
申请人 LSIP, LLC 发明人 HOHJOH, HIROHIKO;TAKAHASHI, MASAKI
分类号 C12N15/11;A61K31/7105;A61P35/00;A61P35/02;C12N15/113 主分类号 C12N15/11
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