- 专利标题: RNA-INTERFERENCE-INDUCING NUCLEIC ACID MOLECULE ABLE TO PENETRATE INTO CELLS, AND USE THEREFOR
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申请号: EP13794539.0申请日: 2013-05-21
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公开(公告)号: EP2853597B1公开(公告)日: 2018-12-26
- 发明人: HONG, Sun Woo
- 申请人: Olix Pharmaceuticals, Inc.
- 申请人地址: Room 1305, Star Valley 99 Digital-ro 9-gil Geumcheon-gu Seoul 153-777 KR
- 专利权人: Olix Pharmaceuticals, Inc.
- 当前专利权人: Olix Pharmaceuticals, Inc.
- 当前专利权人地址: Room 1305, Star Valley 99 Digital-ro 9-gil Geumcheon-gu Seoul 153-777 KR
- 代理机构: Ladendorf, Oliver
- 优先权: KR20120053950 20120522
- 国际公布: WO2013176477 20131128
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; C12N15/63 ; A61K48/00
摘要:
The present invention relates to a novel, RNAi-inducing nucleic acid molecule having cell penetrating ability and the use thereof, and more particularly, to a novel, RNAi-inducing double-stranded nucleic acid molecule, which has a replacement of the phosphate backbone of at least one nucleotide with phosphorothioate or phosphorodithioate, and has a lipophilic compound conjugated thereto, and thus has high target gene-silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle, and to a method of silencing a target gene using the nucleic acid molecule. The nucleic acid structure according to the present invention has both cholesterol modification and phosphorothioate modification introduced therein, and thus has high gene silencing efficiency while having the ability to penetrate cells without needing a separate intracellular delivery vehicle. Thus, it can be delivered into an actual target area in an amount sufficient for induction of RNAi, and thus can overcome the in vivo delivery problem occurring in the prior art. Therefore, the nucleic acid molecule according to the invention can effectively substitute for conventional siRNA molecules to treat cancer or viral infections.
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