DRUMMOND-SAMUELSON, MEGHAN,HAINES, JEFFERY,HARTFORD, SUZANNE,FRENDEWEY, DAVID,ZAMBROWICZ, BRIAN,MURPHY, ANDREW J.
申请号：
CA3071712
公开号：
CA3071712A1
申请日:
2018.09.28
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus and methods of using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus express a human transthyretin protein or a chimeric transthyretin protein, fragments of which are from human transthyretin. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR. Methods are also provided for making such non-human animals comprising a humanized TTR locus.
