发明申请
US20090149407A1 RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER'S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA)
有权
使用短暂干扰核酸(siNA)的RNA干扰介导的治疗阿尔茨海默病
- 专利标题: RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER'S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA)
- 专利标题(中): 使用短暂干扰核酸(siNA)的RNA干扰介导的治疗阿尔茨海默病
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申请号: US12170290申请日: 2008-07-09
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公开(公告)号: US20090149407A1公开(公告)日: 2009-06-11
- 发明人: James McSwiggen , Leonid Beigelman
- 申请人: James McSwiggen , Leonid Beigelman
- 申请人地址: US CA San Francisco
- 专利权人: Sirna Therapeutics, Inc.
- 当前专利权人: Sirna Therapeutics, Inc.
- 当前专利权人地址: US CA San Francisco
- 主分类号: A61K31/7105
- IPC分类号: A61K31/7105 ; C07H21/02
摘要:
The present invention concerns methods and reagents useful in modulating BACE gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against beta-secretase (BACE), amyloid precursor protein (APP), pin-1, presenillin 1 (PS-1) and/or presenillin 2 (PS-2) gene expression and/or activity. The small nucleic acid molecules are useful in the treatment of Alzheimer's disease and any other condition that responds to modulation of BACE, APP, pin-1, PS-1 and/or PS-2 expression or activity.
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