发明名称 |
PCSK9遺伝子の発現を阻害するための組成物および方法 |
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. |
申请公布号 |
JP6034316(B2) |
申请公布日期 |
2016.11.30 |
申请号 |
JP20140018817 |
申请日期 |
2014.02.03 |
申请人 |
アルナイラム ファーマシューティカルズ, インコーポレイテッドALNYLAM PHARMACEUTICALS, INC. |
发明人 |
パメラ タン;バージット ブラムラーゲ;マリア フランク−カーメネツスキ;ケビン フィッツジェラルド;アキン アキニック;ビクター イー. コテリャンスキ |
分类号 |
C12N15/113;A61K31/7088;A61K48/00;A61P3/06;C12N5/10 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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