A liver-humanized mouse model of carbamoyl phosphate synthetase 1-deficiency

Raghuraman C Srinivasan, Mihaela Zabulica, Christina Hammarstedt, Tingting Wu, Roberto Gramignoli, Kristina Kannisto, Ewa Ellis, Ahmad Karadagi, Ralph Fingerhut, Gabriella Allegri, Véronique Rüfenacht, Beat Thöny (+3 others)
2019
A liver-humanized mouse model for CPS1-deficiency was generated by the high-level repopulation of the mouse liver with CPS1-deficient human hepatocytes. When compared with mice that are highly repopulated with CPS1-proficient human hepatocytes, mice that are repopulated with CPS1deficient human hepatocytes exhibited characteristic symptoms of human CPS1 deficiency including an 80% reduction in CPS1 metabolic activity, delayed clearance of an ammonium chloride infusion, elevated
doi:10.5167/uzh-217459 fatcat:kcqh66vdkfhcjmgjunpu5rvu44