Alexander O. Mujica,Viktoria Gusarova,Cheng Wang,Christos Kyratsous,Terra Potocky,Katherine Cygnar,Joel Martin
申请号：
US16625168
公开号：
US20200154684A1
申请日:
2018.06.27
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Non-human animal cells and non-human animals comprising a humanized Asgr1 locus and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized Asgr1 locus express a human ASGR1 protein or an Asgr1 protein, fragments of which are from human ASGR1. Methods are provided for using such non-human animals comprising a humanized Asgr1 locus to assess in vivo efficacy of human-ASGR1-mediated delivery of therapeutic molecules or therapeutic complexes to the liver and to assess the efficacy of therapeutic molecules or therapeutic complexes acting via human-ASGR1-mediated mechanisms.
