Invention Grant
US08314075B2 Compositions and methods for inhibiting expression of huntingtin gene
有权
抑制亨廷忌汀基因表达的组合物和方法
- Patent Title: Compositions and methods for inhibiting expression of huntingtin gene
- Patent Title (中): 抑制亨廷忌汀基因表达的组合物和方法
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Application No.: US13252917Application Date: 2011-10-04
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Publication No.: US08314075B2Publication Date: 2012-11-20
- 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: Alynylam Pharmaceuticals, Inc.
- Current Assignee: Alynylam Pharmaceuticals, Inc.
- Current Assignee Address: US MA Cambridge
- Agency: Sorell, Leanna & Schmidt, LLP.
- Agent William D. Schmidt, Esq.
- Main IPC: A61K31/70
- IPC: A61K31/70 ; 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
- US20120095076A1 COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF HUNTINGTIN GENE Public/Granted day:2012-04-19
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