Recapitulating the immune system of hemophilia A patients with inhibitors using immunodeficient mice
Journal
Thrombosis research
Journal Volume
235
Pages
155 - 163
Date Issued
2024-03
Author(s)
Yen, Ching-Tzu
Yang, Yung-Li
Chen, Shu-Huey
Wang, Jiaan-Der
Fan, Meng-Ni
Chen, Li-Fu
Yu, I-Shing
Tsai, Dong-Yan
Lin, Kuo-I
Tao, Mi-Hua
Abstract
Treating hemophilia A patients who develop inhibitors remains a clinical challenge. A mouse model of hemophilia A can be used to test the efficacy of strategies for inhibitor suppression, but the differences in the immune systems of mice and humans limit its utility. To address this shortcoming, we established a humanized NOD/SCID-IL2rγnull hemophilia A (hu-NSG-HA) mouse model with a severely deficient mouse immune system presenting a patient's adapted immune cells.
Subjects
FVIII inhibitor; Hemophilia A; Humanized hemophilia NSG mice
SDGs
Type
journal article