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KNOCK-IN MOUSE FOR MODELLING BLOCKADE OF HUMAN TNFALPHA

机译:用于模拟人TNFα封锁的敲入鼠标

摘要

The invention relates to a transgenic mouse, wherein the complete endogenous murine TNFα-gene including its upstream regulatory sequences is replaced by the human homologous TNFα-gene including its upstream regulatory sequences or a mutant thereof, wherein the mutant is characterized by deletion, addition, substitution, and/or inversion of one or more nucleotides so that the functional properties of the modified human TNFα-gene are conserved and cells, tissues and cell lines derived thereof. The invention further relates to an in-vivo method of screening, investigating and/or preclinical testing of compounds and/or drug candidates for human anti-TNFα therapy using said transgenic mouse and an analogous method using the cells, tissues and cell lines derived thereof.
机译:本发明涉及一种转基因小鼠,其中包括其上游调节序列的完整内源鼠TNFα基因被包括其上游调节序列的人同源TNFα基因或其突变体替代,其中该突变体的特征在于缺失,添加,一个或多个核苷酸的取代和/或倒置,从而使修饰的人TNFα基因的功能特性得以保留,并衍生出其细胞,组织和细胞系。本发明还涉及使用所述转基因小鼠筛选,研究和/或临床前测试用于人抗TNFα疗法的化合物和/或候选药物的体内方法,以及使用其衍生的细胞,组织和细胞系的类似方法。 。

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