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Non-human animal containing humanized TRKB locus

机译:包含人源化TRKB基因座的非人类动物

摘要

Provided are non-human animal genomes comprising humanized TRKB loci, non-human animal cells, and non-human animals and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals. A non-human animal cell or non-human animal comprising a humanized TRKB locus expresses a chimeric transthyretin protein whose human TRKB protein or fragment thereof is derived from human TRKB. Methods of using such non-human animals, including humanized TRKB loci, are provided to assess the in vivo efficacy of human-TRKB-targeting reagents, such as nuclease agonists designed to target human TRKB.
机译:提供的是包含人源化 TRKB 基因座,非人类动物细胞和非人类动物的非人类动物基因组,以及制备和使用此类非人类动物基因组的方法,非人类动物细胞和非人类动物。包含人源化的 TRKB 基因座的非人动物细胞或非人动物表达嵌合运甲状腺素蛋白蛋白,其人TRKB蛋白或其片段衍生自人TRKB。提供了使用包括人源化的 TRKB 基因座在内的此类非人类动物的方法,以评估靶向人类TRKB的试剂(例如设计为靶向人类TRKB的核酸酶激动剂)的体内功效。

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