siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS

更新时间:2023-05-21 17:07:07 阅读: 评论:0

专利名称:siRNAs WITH AT LEAST TWO LIGANDS ATwell
DIFFERENT ENDS
ibelieve什么意思发明人:Lucas Bethge,Adrien Weingärtner,Judith
Hauptmann
brow申请号:US17045125
申请日:20190405孔雀东南飞剧本
duplex公开号:US20210155926A1
uniform公开日:
look down>研究生报考类别20210527
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专利附图:
摘要:There is provided inter alia a conjugate for inhibiting expression of a target gene in a cell, said conjugate comprising a nucleic acid portion and ligand portions, said nucleic
acid portion comprising at least one duplex region that compris at least a portion of a first RNA strand and at least a portion of a cond RNA strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene, said ligand portions comprising a linker moiety and a targeting ligand for in vivo targeting of cells and being conjugated exclusively to the 3′ and/or 5′ ends of one or both RNA strands, wherein the 5′ end of the first RNA strand is not conjugated, wherein: (i) the cond RNA strand is conjugated at the 5′ end to the targeting ligand, and wherein (a) the cond RNA strand is also conjugated at the 3′ end to the targeting ligand and the 3′end of the first RNA strand is not conjugated; or (b) the first RNA strand is conjugated at the 3′ end to the targeting ligand and the 3′ end of the cond RNA strand is not conjugated; or (c) both the cond RNA strand and the first RNA strand are also conjugated at the 3′ ends to the targeting ligand; or (ii) both the cond RNA strand and the first RNA strand are conjugated at the 3′ ends to the targeting ligand and the 5′ end of the cond RNA strand is not conjugated.
申请人:SILENCE THERAPEUTICS GMBH
地址:Berlin DE郑州英语翻译
国籍:DE
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