Publication Type Review
Authors Chiuchiolo M, Crystal R
Journal Ann Am Thorac Soc
Volume 13 Suppl 4
Issue Suppl 4
Pagination S352-69
Date Published 08/01/2016
ISSN 2325-6621
Keywords Genetic Therapy, Pulmonary Emphysema, Serine Proteinase Inhibitors, alpha 1-Antitrypsin, alpha 1-Antitrypsin Deficiency
Abstract 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 contamination and allergic reactions, and is costly. As an alternative, gene therapy offers the advantage of single administration, eliminating the burden of protein infusion, and reduced risks and costs. The focus of this review is to describe the various strategies for AAT gene therapy for the pulmonary manifestations of AAT deficiency and the state of the art in bringing AAT gene therapy to the bedside.
DOI 10.1513/AnnalsATS.201506-344KV
PubMed ID 27564673
PubMed Central ID PMC5059492
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