发明名称 |
Compositions and methods for inhibiting expression of Huntingtin gene |
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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申请公布号 |
US7320965(B2) |
申请公布日期 |
2008.01.22 |
申请号 |
US20060588674 |
申请日期 |
2006.10.27 |
申请人 |
ALNYLAM PHARMACEUTICALS, INC. |
发明人 |
SAH DINAH WEN-YEE;HADWIGER PHILIPP;ROEHL INGO;BRAMLAGE BIRGIT;TAN PAMELA;VORNLOCHER HANS-PETER;BUMCROT DAVID |
分类号 |
A61K31/70;C07H21/04;C12N5/00;C12N5/02;C12P19/34;C12Q1/68 |
主分类号 |
A61K31/70 |
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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