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Article

Adeno-associated Virus-mediated Silencing of Sox4 Leads to Long-Term Amelioration of Liver Phenotypes in Mouse Models of Alagille Syndrome.

Gastroenterology - 1 Oct 2025

Fox Duncan, Xie Jun, Burwinkel Jennifer L, Adams Josh M, Chetal Kashish, Keivandarian Marzieh, Faingelernt Yaniv, Subramanian Sanjay, Lopez Mario F, Peters Anna L, Salomonis Nathan, Zarrin-Khameh Neda, Gao Guangping, Huppert Stacey S, Jafar-Nejad Hamed

Abstract excerpt

BACKGROUND & AIMS: In patients with Alagille syndrome (ALGS), bile duct paucity often leads to severe cholestatic phenotypes for which liver transplantation remains the only definitive treatment. No Food and Drug Administration-approved mechanism-based strategies exist to enhance biliary development in ALGS or other diseases with bile duct paucity. We aimed to identify a therapeutic target to address this unmet...

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Share to Alagille Syndrome
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 40446945
DOI: 10.1053/j.gastro.2025.04.033
PMCID: PMC12354214

Genes

SOX4

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

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