Invention Grant
US07994309B2 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
- Patent Title: Compositions and methods for inhibiting expression of a mutant gene
- Patent Title (中): 用于抑制突变基因表达的组合物和方法
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Application No.: US12817009Application Date: 2010-06-16
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Publication No.: US07994309B2Publication Date: 2011-08-09
- Inventor: Roland Kreutzer , Stefan Limmer , Matthias John
- Applicant: Roland Kreutzer , Stefan Limmer , Matthias John
- Applicant Address: US MA Cambridge
- Assignee: Alnylam Pharmaceuticals, Inc.
- Current Assignee: Alnylam Pharmaceuticals, Inc.
- Current Assignee Address: US MA Cambridge
- Agency: Fenwick & West LLP
- Priority: DE10155280 20011026; DE10158411 20011129; DE10160151 20011207; DE10235620 20020802
- Main IPC: C07H21/04
- IPC: C07H21/04

Abstract:
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.
Public/Granted literature
- US20100291194A1 Compositions and Methods for Inhibiting Expression of a Mutant Gene Public/Granted day:2010-11-18
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