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Article

Targeting intracellular degradation pathways for treatment of liver disease caused by α1-antitrypsin deficiency.

Pediatric research - 1 Jan 2014

Wang Yan, Perlmutter David H

Abstract excerpt

The classic form of α1-antitrypsin deficiency (ATD) is a well-known genetic cause of severe liver disease in childhood. A point mutation alters the folding of a hepatic secretory glycoprotein such that the protein is prone to misfolding and polymerization. Liver injury, characterized predominantly by fibrosis/cirrhosis and carcinogenesis, is caused by the proteotoxic effect of polymerized mutant α1-antitrypsin Z...

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Identifiers

PMID: 24226634
DOI: 10.1038/pr.2013.190
PMCID: PMC4174576

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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