发明授权
US08273870B2 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
-
申请号： US13177316申请日： 2011-07-06
-
公开(公告)号： US08273870B2公开(公告)日： 2012-09-25
- 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： C07H21/04
- IPC分类号： C07H21/04
摘要：
The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a mutant gene, comprising a first strand that has a complementary region that is complementary to at least a portion of an RNA transcript of at least part of the mutant target gene and a second strand of the dsRNA complementary or substantially complementary to the first strand. 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.
公开/授权文献
信息查询
