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TDP-43 KNOCK-IN MOUSE MODEL OF AMYOTROPHIC LATERAL SCLEROSIS

机译：TDP-43肌萎缩侧索硬化症的敲入小鼠模型

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摘要

The invention relates to a genetically modified mouse comprising a heterozygous mutation of Tardbp (TDP-43) gene in that the Asn at amino acid 390 in TDP-43 is substituted with an amino acid that is different from Asn, wherein the genetically modified mouse exhibits Amyotrophic lateral sclerosis (ALS)-like phenotypes, TDP-43 proteinopathies and/or motor neuron degeneration. The invention also so relates to an isolated spinal cord motor neuron differentiated from an embryonic stem cell (ESC) that is obtained from an offspring of a genetically modified mouse according to the invention. Methods for identifying an agent alleviating and/or suppressing ALS-TDP pathogenesis are also disclosed.

机译：本发明涉及包含Tardbp（TDP-43）基因的杂合突变的转基因小鼠，其中TDP-43中390位氨基酸的Asn被不同于Asn的氨基酸取代，其中所述转基因小鼠表现出肌萎缩性侧索硬化症（ALS）型，TDP-43蛋白病和/或运动神经元变性。本发明还因此涉及从胚胎干细胞（ESC）分化的分离的脊髓运动神经元，所述胚胎干细胞从根据本发明的基因修饰小鼠的后代获得。还公开了鉴定减轻和/或抑制ALS-TDP发病机理的试剂的方法。

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公开/公告号US2019335727A1

专利类型
公开/公告日2019-11-07

原文格式PDF
申请/专利权人 ACADEMIA SINICA;
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申请/专利号US201916387430
发明设计人 CHE-KUN JAMES SHEN;SHIH-LING HUANG;
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申请日2019-04-17
分类号A01K67/027;C07K14/47;C12N5/0793;G01N33/564;A61K49;G01N33/50;
国家 US
入库时间 2022-08-21 12:08:53

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