A humanized mouse model of APOB deficiency recapitulates Hypobetalipoproteinemia.

Authors

Amita Tiyaboonchai, Anne Vonada, Jeffrey Posey, Kevin L Keys, Carl Pelz, Helen Chen, Xiping Cheng, Markus Grompe

Published in

Journal of lipid research. Pages 101142. Sep 02, 2026. Epub Sep 02, 2026.

Abstract

Lipoprotein metabolism is significantly different between mice and humans thus making it difficult to model disorders of human lipid metabolism in transgenic mice. Systemic lipoprotein metabolism is predominantly governed by hepatocytes, and mice with humanized livers display human-like lipid profiles. Here we report a highly efficient method to knock out genes in human hepatocytes while retaining their ability to repopulate immune deficient rodents. As proof-of-principle Fah deficient, immune compromised mice were repopulated with Apolipoprotein B (APOB) knockout human hepatocytes. Mice humanized with knockout cells recapitulated typical features of human hypobetalipoproteinemia. We conclude that at least some human lipid metabolism disorders can be modeled in liver chimeric mice using human knockout hepatocytes.

PMID:
42686063
Bibliographic data and abstract were imported from PubMed on 03 Sep 2026.

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