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Article

Novel treatment strategies for liver disease due to α1-antitrypsin deficiency.

Clinical and translational science - 1 Jun 2012

Maurice Nicholas, Perlmutter David H

Abstract excerpt

Alpha1-antitrypsin (AT) deficiency is the most common genetic cause of liver disease in children and is also a cause of chronic hepatic fibrosis, cirrhosis, and hepatocellular carcinoma in adults. Recent advances in understanding how mutant AT molecules accumulate within hepatocytes and cause liver cell injury have led to a novel strategy for chemoprophylaxis of this liver disease. This strategy involves a class...

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Identifiers

PMID: 22686209
DOI: 10.1111/j.1752-8062.2011.00363.x
PMCID: PMC3982223

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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