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Intrapleural Gene Therapy for Alpha-1 Antitrypsin Deficiency-Related Lung Disease

1 Jan 2018

Abstract excerpt

Alpha-1 antitrypsin deficiency (AATD) manifests primarily as early-onset emphysema caused by the destruction of the lung by neutrophil elastase due to low amounts of the serine protease inhibitor alpha-1 antitrypsin (AAT). The current therapy involves weekly intravenous infusions of AAT-derived from pooled human plasma that is efficacious, yet costly. Gene therapy applications designed to provide constant levels...

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PMID: 30723782
DOI: 10.15326/jcopdf.5.4.2017.0160

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