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1. 发明申请

WO2005044196A2 NOVEL MODULATORS OF AMYLOID-BETA PRODUCTION AND USES THEREOF 审中-公开
标题翻译： AMYLOID-BETA生产的新型调节剂及其用途
公开(公告)号：WO2005044196A2
公开(公告)日：2005-05-19
申请号：PCT/US2004036459
申请日：2004-11-01
申请人： UNIV COLUMBIA , KIM TAE-WAN , LEE HAHN-JUN
发明人： KIM TAE-WAN , LEE HAHN-JUN
IPC分类号： A61K38/00 , C07H21/04 , C07K14/47 , C12Q1/68 , A61K
CPC分类号： C07H21/04 , A61K38/00 , C07K14/4702 , C12Q1/6883
摘要： The present invention provides isolated nucleic acid sequences encoding presenilin stabilization factor (PSF) and PSF-like (PSFL) protein, vectors comprising same, host cells transformed with the vectors, and transgenic animals containing the host cells. The present invention further provides purified PSF and PSFL polypeptides, methods for making same, and pharmaceutical compositions comprising the polypeptides. Also provided are agents reactive with the nucleic acid sequences and polypeptides, kits comprising same, and methods for producing same. The present invention further provides methods for decreasing amyloid-beta (Aß) production, destabilizing presenilin or nicastrin, destabilizing a gamma-secretase complex, and inhibiting activity of gamma-secretase, and pharmaceutical compositions for accomplishing same. The present invention further provides methods for treating neurodegeneration in a subject. Finally, the present invention provides an in vitro system for identifying an agent that modulates production of Aß or an Aß precursor, methods for making and using same, and agents identified by same.
摘要翻译：本发明提供编码早老蛋白稳定因子（PSF）和PSF样（PSFL）蛋白的分离的核酸序列，包含其的载体，用载体转化的宿主细胞和含有宿主细胞的转基因动物。本发明还提供了纯化的PSF和PSFL多肽，其制备方法以及包含多肽的药物组合物。还提供了与核酸序列和多肽反应的试剂，包含其的试剂盒及其制备方法。本发明还提供降低淀粉样蛋白β（Aß）产生，去稳定早老蛋白或奈米卡品，去稳定γ-分泌酶复合物和抑制γ-分泌酶活性的方法和用于实现γ-分泌酶的药物组合物的方法。本发明还提供了治疗受试者神经变性的方法。最后，本发明提供了用于鉴定调节Aβ或Aβ前体的生产的试剂的体外系统，用于制备和使用它们的方法以及由其鉴定的试剂。

2. 发明申请

WO2005044196A3 NOVEL MODULATORS OF AMYLOID-BETA PRODUCTION AND USES THEREOF 审中-公开
公开(公告)号：WO2005044196A3
公开(公告)日：2005-05-19
申请号：PCT/US2004/036459
申请日：2004-11-01
申请人： THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK , KIM, Tae-Wan , LEE, Hahn-Jun
发明人： KIM, Tae-Wan , LEE, Hahn-Jun
IPC分类号： C07H21/02 , C07H21/04 , C07K14/435 , C07K16/18 , C12N15/63 , A01K67/00 , C12Q1/68 , A61K38/00 , A61K31/713
摘要： The present invention provides isolated nucleic acid sequences encoding presenilin stabilization factor (PSF) and PSF-like (PSFL) protein, vectors comprising same, host cells transformed with the vectors, and transgenic animals containing the host cells. The present invention further provides purified PSF and PSFL polypeptides, methods for making same, and pharmaceutical compositions comprising the polypeptides. Also provided are agents reactive with the nucleic acid sequences and polypeptides, kits comprising same, and methods for producing same. The present invention further provides methods for decreasing amyloid-beta (Aß) production, destabilizing presenilin or nicastrin, destabilizing a gamma-secretase complex, and inhibiting activity of gamma-secretase, and pharmaceutical compositions for accomplishing same. The present invention further provides methods for treating neurodegeneration in a subject. Finally, the present invention provides an in vitro system for identifying an agent that modulates production of Aß or an Aß precursor, methods for making and using same, and agents identified by same.

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