发明公开
EP2584048A1 Compositions and methods for inhibiting expression of the PCSK9 gene
有权
用于抑制PCSK9基因表达的组合物和方法
- 专利标题： Compositions and methods for inhibiting expression of the PCSK9 gene
- 专利标题（中）： 用于抑制PCSK9基因表达的组合物和方法
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申请号： EP12178667.7申请日： 2007-05-10
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公开(公告)号： EP2584048A1公开(公告)日： 2013-04-24
- 发明人: Tan, Pamela , Bramlage, Birgit , Frank-Kamenetsky, Maria , Fitzgerald, Kevin , Akinc, Akin , Kotelianski, Victor, E.
- 申请人： Alnylam Pharmaceuticals Inc.
- 申请人地址： 300 Third Street Cambridge, MA 02142 US
- 专利权人： Alnylam Pharmaceuticals Inc.
- 当前专利权人： Alnylam Pharmaceuticals Inc.
- 当前专利权人地址： 300 Third Street Cambridge, MA 02142 US
- 代理机构： Vossius & Partner
- 优先权： US799458P 20060511; US817203P 20060627; US840089P 20060825; US829914P 20061018; US901134P 20070213
- 主分类号： C12Q1/68
- IPC分类号： C12Q1/68 ; A01N43/04 ; C07H21/04 ; A61K31/07 ; C12N15/113
摘要：
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a human PCSK9 gene in a cell, wherein said dsRNA comprises at least two sequences that are complementary to each other and wherein a sense strand comprises a first sequence and an antisense strand comprises a second sequence comprising a region of complementarity which is substantially complementary to at least a part of a mRNA encoding PCSK9, and wherein said region of complementarity is less than 30 nucleotides in length and wherein said dsRNA, upon contact with a cell expressing said PCSK9, inhibits expression of said PCSK9 gene, wherein (a) said first sequence is the sequence of SEQ ID NO: 1229 and said second sequence is the sequence of SEQ ID NO: 1230; (b) said first sequence is the sequence of SEQ ID NO: 1227 and said second sequence is the sequence of SEQ ID NO: 1228; or (c) said part of said mRNA encoding PCSK9 is the sequence from position 3530 to 3548 of the mRNA sequence of database entry NM_174936.
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