发明名称 RNA interference mediated treatment of alzheimer's disease using short interfering nucleic acid (siNA)
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.
申请公布号 US8017765(B2) 申请公布日期 2011.09.13
申请号 US20090640411 申请日期 2009.12.17
申请人 MERCK SHARP & DOHME CORP. 发明人 MCSWIGGEN JAMES;BEIGELMAN LEONID
分类号 C07H21/04;A61K38/00;C12N15/11;C12N15/113;C12N15/115;C12N15/87 主分类号 C07H21/04
代理机构 代理人
主权项
地址