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