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IUBMB Life. 2009 Jan;61(1):1-5. doi: 10.1002/iub.127.

Serpin polymerization and its role in disease--the molecular basis of alpha1-antitrypsin deficiency.

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  • 1Department of Biochemistry and Molecular Biology, Monash University, Melbourne, Victoria, Australia.

Abstract

Protein aggregation is the cause of several human diseases. Understanding the molecular mechanisms involved in protein aggregation requires knowledge of the kinetics and structures populated during the reaction. Arguably, the best structurally characterized misfolding reaction is that of alpha(1)-antitrypsin. Alpha(1)-antitrypsin misfolding leads to both liver disease and emphysema and affect approximately 1 in 2000 of the population. This review will focus on the mechanism of alpha(1)-antitrypsin misfolding and the development of potential therapeutic strategies.

PMID:
18785256
[PubMed - indexed for MEDLINE]
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