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Complement factor H gene knockout rat as a model of C3 glomerulopathy

机译:补体因子H基因敲除RAT作为C3肾小球疗法模型

摘要

Methods for producing and using rat cells and rats including deactivated CFH loci, and such rat cells and rats are provided.Rats with an inactivated CFH locus model C3 glomerulopathy (C3G).A method for using such rats including an inactivated CFH locus is provided to assess in vivo efficacy of the putative C3G therapeutic agent.The present invention provides gene modified rats, including, for example, an inactivated endogenous CFH locus.
机译:提供制备和使用包括失活CFH基因座的大鼠的方法和大鼠的大鼠,以及这种大鼠细胞和大鼠的大鼠。用灭活的CFH基因座模型C3肾小球疗法(C3G)。使用包括灭活CFH基因座的这种大鼠的方法 评估推定的C3G治疗剂的体内疗效。本发明提供了基因改性大鼠,包括例如灭活的内源性CFH基因座。

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