Humanized liver mouse models in preclinical drug development: current status, translation challenges and emerging technologies
Humanized liver mouse models are constructed by engrafting primary human hepatocytes into immunodeficient mouse livers, thereby reconstructing human-specific metabolic and transport pathways and significantly improving the prediction of pharmacokinetics, drug interactions and hepatotoxicity. These models have been widely applied to MASLD/MASH, fibrosis, hepatocellular carcinoma and viral hepatitis research but remain constrained by the absence of intestinal CYP3A4 expression and functional immune compartments. Emerging approaches, including dual humanization, non-parenchymal cell supplementation, vascularized 3D bioprinting and application of patient-derived hepatocytes, combined with advanced in vitro and computational platforms, further strengthen translational fidelity, reduce R&D attrition and expand the scope for modeling complex metabolism-related diseases.
Authors
- Xumiao Zhang
- Yuanbin Song
- Lu Lu
- wei Xiao
Institutions
- Sun Yat-sen University (CN)
- The Seventh Affiliated Hospital of Sun Yat-sen University (CN)
- Sun Yat-sen University Cancer Center (CN)
- State Key Laboratory of Oncology in South China
Publication Details
- Journal
- Drug Discovery Today
- Published
- 2026-01-21
- DOI
- https://doi.org/10.1016/j.drudis.2026.104611
- Primary Topic
- Liver physiology and pathology
- Type
- article
- Field-Weighted Citation Impact
- 0.00