发明授权
- 专利标题: Compositions and methods for inhibiting expression of factor V
- 专利标题(中): 抑制因子V表达的组合物和方法
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申请号: US13010300申请日: 2011-01-20
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公开(公告)号: US08658782B2公开(公告)日: 2014-02-25
- 发明人: Hans-Peter Vornlocher , John M. Maraganore
- 申请人: Hans-Peter Vornlocher , John M. Maraganore
- 申请人地址: US MA Cambridge
- 专利权人: Alynylam Pharmaceuticals, Inc.
- 当前专利权人: Alynylam Pharmaceuticals, Inc.
- 当前专利权人地址: US MA Cambridge
- 代理机构: Fenwik & West LLP
- 主分类号: C07H21/04
- IPC分类号: C07H21/04 ; C12N15/11 ; C12N5/00 ; C12N15/00 ; C12Q1/68
摘要:
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Factor V Leiden mutant gene (Factor V Leiden mutant gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Factor V Leiden mutant gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Factor V Leiden mutant gene using the pharmaceutical composition; and methods for inhibiting the expression of the Factor V Leiden mutant gene in a cell.
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