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Human iPSC-derived liver organoids model multicellular tissue responses and therapeutic rescue in Wolman disease

2025-12-22

Abstract excerpt

Wolman disease (WD), the severe infantile form of lysosomal acid lipase deficiency, is a rare metabolic disorder caused by inactivating mutations in the LIPA gene. Although WD is characterized by profound hepatic dysfunction, experimental human systems capable of modelling multicellular liver pathology and supporting therapeutic testing remain limited. Here, we generated an isogenic human model of WD by introducin...

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Literature Corpus work
af83de13-2293-522f-ba3a-893ddd8742e2
DOI
10.64898/2025.12.16.694623
Open publication

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Human iPSC-derived liver organoids model multicellular tissue responses and therapeutic rescue in Wolman diseaseDOI 10.64898/2025.12.16.694623
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