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.

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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
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article

Humanized liver mouse models in preclinical drug development: current status, translation challenges and emerging technologies

Xumiao Zhang, Yuanbin Song, Lu Lu, wei Xiao
Drug Discovery Today
Liver physiology and pathology
article

Humanized liver mouse models in preclinical drug development: current status, translation challenges and emerging technologies

Xumiao Zhang, Yuanbin Song, Lu Lu, wei Xiao
article en

Abstract

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.

Drug Discovery TodayVol. 31(2)
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
Openalex Percentile: Top 80%
Liver physiology and pathology
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