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Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease

1 Aug 2016

Abstract excerpt

Alpha-1 antitrypsin (AAT) deficiency, characterized by low plasma levels of the serine protease inhibitor AAT, is associated with emphysema secondary to insufficient protection of the lung from neutrophil proteases. Although AAT augmentation therapy with purified AAT protein is efficacious, it requires weekly to monthly intravenous infusion of AAT purified from pooled human plasma, has the risk of viral...

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PMID: 27564673
DOI: 10.1513/annalsats.201506-344kv

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