发明授权
US07763590B2 Compositions and methods for inhibiting expression of a mutant gene
有权
用于抑制突变基因表达的组合物和方法
- 专利标题： Compositions and methods for inhibiting expression of a mutant gene
- 专利标题（中）： 用于抑制突变基因表达的组合物和方法
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申请号： US10384463申请日： 2003-03-07
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公开(公告)号： US07763590B2公开(公告)日： 2010-07-27
- 发明人: Roland Kreutzer , Stefan Limmer , Matthias John
- 申请人： Roland Kreutzer , Stefan Limmer , Matthias John
- 申请人地址： US MA Cambridge
- 专利权人： Alnylam Pharmaceuticals, Inc.
- 当前专利权人： Alnylam Pharmaceuticals, Inc.
- 当前专利权人地址： US MA Cambridge
- 代理机构： Fenwick & West LLP
- 优先权： DE10155280 20011026; DE10158411 20011129; DE10160151 20011207; DE10235620 20020802
- 主分类号： A61K48/00
- IPC分类号： A61K48/00
摘要：
The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a complementary RNA strand having a complementary region that is substantially complementary to a portion of the mutant gene, and which is partially complementary to the corresponding wild-type gene. The invention further relates to a pharmaceutical composition comprising the dsRNA and a pharmaceutically acceptable carrier. The pharmaceutical compositions are useful for inhibiting the expression of a target mutant gene, as well as for treating diseases caused by expression of the target gene. The invention also relates to methods for inhibiting the expression of a target mutant gene, as well as methods for treating diseases caused by the expression of the target gene.