TY - JOUR T1 - α<sub>1</sub>-Antitrypsin deficiency • 4: Molecular pathophysiology JF - Thorax JO - Thorax SP - 529 LP - 535 DO - 10.1136/thx.2003.006528 VL - 59 IS - 6 AU - D A Lomas AU - H Parfrey Y1 - 2004/06/01 UR - http://thorax.bmj.com/content/59/6/529.abstract N2 - 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. ER -