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Thorax. 2004 June; 59(6): 529–535.
PMCID: PMC1747018

α1-Antitrypsin deficiency • 4: Molecular pathophysiology


The molecular basis of α1-antitrypsin deficiency is reviewed and is shown to be due to the accumulation of mutant protein as ordered polymers within the endoplasmic reticulum of hepatocytes. The current goals are to determine the cellular response to polymeric α1-antitrypsin and to develop therapeutic strategies to block polymerisation in vivo.

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