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Article

Knockdown of Z Mutant Alpha-1 Antitrypsin In Vivo Using Modified DNA Antisense Oligonucleotides.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2017

Aghajan Mariam, Guo Shuling, Monia Brett P

Abstract excerpt

Alpha-1 antitrypsin (AAT) is a serum protease inhibitor, mainly expressed in and secreted from hepatocytes, important for regulating neutrophil elastase activity among other proteases. Various mutations in AAT cause alpha-1 antitrypsin deficiency (AATD), a rare hereditary disorder that results in liver disease due to accumulation of AAT aggregates and lung disease from excessive neutrophil elastase activity. PiZ...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 28752452
DOI: 10.1007/978-1-4939-7163-3_12

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