Invention Grant
US08080532B2 Compositions and methods for inhibiting expression of Huntingtin gene
有权
抑制Huntingtin基因表达的组成和方法
- Patent Title: Compositions and methods for inhibiting expression of Huntingtin gene
- Patent Title (中): 抑制Huntingtin基因表达的组成和方法
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Application No.: US12417502Application Date: 2009-04-02
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Publication No.: US08080532B2Publication Date: 2011-12-20
- Inventor: Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
- Applicant: Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
- Applicant Address: US MA Cambridge
- Assignee: Alnylam Pharmaceuticals, Inc.
- Current Assignee: Alnylam Pharmaceuticals, Inc.
- Current Assignee Address: US MA Cambridge
- Agency: Fenwick & West LLP
- Main IPC: C12N15/11
- IPC: C12N15/11 ; C07H21/02 ; C07H21/04

Abstract:
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.
Public/Granted literature
- US20100298405A1 Compositions And Methods For Inhibiting Expression Of Huntingtin Gene Public/Granted day:2010-11-25
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