首页> 外国专利> TRANSGENIC RAT AND THE USE THEREOF IN THE ANIMAL MODEL FOR HUMAN HUNTINGDON'S DISEASE AND NUCLEAR CONSTRUCTS, VECTORS AND CELLS FOR THE PRODUCTION THEREOF

TRANSGENIC RAT AND THE USE THEREOF IN THE ANIMAL MODEL FOR HUMAN HUNTINGDON'S DISEASE AND NUCLEAR CONSTRUCTS, VECTORS AND CELLS FOR THE PRODUCTION THEREOF

机译:转基因大鼠及其在动物模型中的应用(人类亨廷顿氏病和核构造,矢量和细胞)

摘要

Huntingdon's Disease (HD) is an autosomal-dominant inherited progredientneurodegenerative disease from the group of CAG repeat/polyglutamine diseasesand is characterized by a triad of psychiatric modifications, dementia andmotory function disorders. On a sub-cellular level, a mutation with extendedCAG trinucleotide repeats has been identified as the cause of Huntingdon'sDisease. The therapeutic effects of certain substances can be tested on theneurochemically indicated transgenic animal models with expanded CAG repeats.In the present invention, transgenic rats were generated and characterized forhuman HD. Said rat model for human HD and other diseases of the CNS includes51 CAG repeats under the control of a rat promoter and has a slowlyprogredient neurological phenotype closely reflecting human HD syndrome. Thecomparability of the rat model in relation to human HD is characterized byneuropathological, neuroradiological and neurochemical modificationsaccompanied by typical behavioural abnormalities.
机译:亨廷顿病(HD)是常染色体显性遗传遗传CAG重复/多谷氨酰胺疾病组的神经退行性疾病其特点是三重性精神病,痴呆和运动功能障碍。在亚细胞水平上,具有延伸的突变CAG三核苷酸重复序列已被确定为亨廷登氏症的病因疾病。某些物质的治疗效果可以在神经化学指示的具有扩大的CAG重复序列的转基因动物模型。在本发明中,转基因大鼠被产生并表征为人类高清。所述用于人类HD和中枢神经系统其他疾病的大鼠模型包括51个CAG在大鼠启动子的控制下重复并缓慢明显反映人类HD综合征的神经系统表型。的与人类HD相关的大鼠模型可比性的特征在于神经病理学,神经放射学和神经化学修饰伴有典型的行为异常。

著录项

  • 公开/公告号CA2523888A1

    专利类型

  • 公开/公告日2003-11-20

    原文格式PDF

  • 申请/专利权人 RIESS OLAF;VON HORSTEN STEPHAN;

    申请/专利号CA20032523888

  • 发明设计人 VON HORSTEN STEPHAN;RIESS OLAF;

    申请日2003-05-14

  • 分类号C12N15/00;A01K67/027;A61K49/00;C07K14/47;C12N5/10;

  • 国家 CA

  • 入库时间 2022-08-21 23:03:55

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