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iPSC-derived hepatocytes accurately recapitulate population diversity in alpha-1-antitrypsin deficiency and offer a novel <i>in vitro</i> model for large-scale drug efficacy screening studies

2025-01-23

Abstract excerpt

<h4>Background</h4> Alpha-1 antitrypsin deficiency (A1ATD) is a hereditary recessive disorder caused by mutations in the SERPINA1 gene. It is a clinically under-recognised disease characterised by low circulating A1AT levels and intracellular accumulation of misfolded A1AT in hepatocytes. Deposition of excessive abnormal A1AT in the liver leads to liver failure, yet no specific treatments are available due to th...

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Literature Corpus work
47991cef-09ef-53fd-bfdd-81dbb545d1fc
DOI
10.1101/2025.01.21.634083
Open publication

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iPSC-derived hepatocytes accurately recapitulate population diversity in alpha-1-antitrypsin deficiency and offer a novel <i>in vitro</i> model for large-scale drug efficacy screening studiesDOI 10.1101/2025.01.21.634083
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