发明申请
- 专利标题: Compositions and Methods for Inhibiting Expression of a Mutant Gene
- 专利标题(中): 抑制突变基因表达的组合物和方法
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申请号: US12817009申请日: 2010-06-16
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公开(公告)号: US20100291194A1公开(公告)日: 2010-11-18
- 发明人: Roland Kreutzer , Stefan Limmer , Matthias John
- 申请人: Roland Kreutzer , Stefan Limmer , Matthias John
- 优先权: DE10155280.7 20011026; DE10158411.3 20011129; DE10160151.4 20011207; DE10235620.3 20020802
- 主分类号: A61K9/127
- IPC分类号: A61K9/127 ; C07H21/02 ; A61K31/713 ; A61K9/50 ; A61K9/51 ; A61K9/00 ; A61P35/00
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.
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