发明授权
US08314075B2 Compositions and methods for inhibiting expression of huntingtin gene
有权
抑制亨廷忌汀基因表达的组合物和方法
- 专利标题： Compositions and methods for inhibiting expression of huntingtin gene
- 专利标题（中）： 抑制亨廷忌汀基因表达的组合物和方法
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申请号： US13252917申请日： 2011-10-04
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公开(公告)号： US08314075B2公开(公告)日： 2012-11-20
- 发明人: Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
- 申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
- 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： Sorell, Leanna & Schmidt, LLP.
- 代理商 William D. Schmidt, Esq.
- 主分类号： A61K31/70
- IPC分类号： A61K31/70 ; C07H21/02 ; C07H21/04
摘要：
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD 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 HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
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