首页> 外国专利> HUNTINGTON DISEASE CELLULAR MODEL: STABLY TRANSFECTED PC12 CELLS EXPRESSING MUTANT HUNTINGTIN

HUNTINGTON DISEASE CELLULAR MODEL: STABLY TRANSFECTED PC12 CELLS EXPRESSING MUTANT HUNTINGTIN

机译:亨廷顿病细胞模型:稳定表达突变亨廷顿蛋白的PC12细胞

摘要

The invention provides compositions and methods for the study, diagnosis, and treatment of Huntington's disease. Compositions comprise stably transfected cell lines that serve as a cellular model for Huntington's disease, sequences corresponding to an antigen associated with Huntington's disease, and monoclonal antibodies that bind specifically to said antigen. The stably transfected cell lines of the invention express truncated mutant huntingtin protein and display cellular defects similar to those observed in cells of patients with Huntington's disease. A method is provided wherein these cell lines are used to screen for therapeutic agents for the treatment of Huntington's disease. The monoclonal antibodies of the invention find use in a diagnostic assay for Huntington's disease.
机译:本发明提供了用于研究,诊断和治疗亨廷顿氏病的组合物和方法。组合物包含用作亨廷顿氏病细胞模型的稳定转染的细胞系,对应于与亨廷顿氏病相关的抗原的序列以及与所述抗原特异性结合的单克隆抗体。本发明的稳定转染的细胞系表达截短的突变亨廷顿蛋白,并显示出类似于在亨廷顿氏病患者的细胞中观察到的细胞缺陷。提供了一种方法,其中这些细胞系用于筛选用于治疗亨廷顿氏病的治疗剂。本发明的单克隆抗体可用于亨廷顿氏病的诊断测定中。

著录项

  • 公开/公告号WO0078813A2

    专利类型

  • 公开/公告日2000-12-28

    原文格式PDF

  • 申请/专利权人 EMORY UNIVERSITY;

    申请/专利号WO2000US16908

  • 发明设计人 LI SHI-HUA;LI XIAO-JIANG;

    申请日2000-06-19

  • 分类号C07K16/00;

  • 国家 WO

  • 入库时间 2022-08-22 01:19:06

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