Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease.

Publication TypeJournal Article
Year of Publication2016
AuthorsChiuchiolo MJ, Crystal RG
JournalAnn Am Thorac Soc
Volume13 Suppl 4
Date Published2016 Aug

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.

Alternate JournalAnn Am Thorac Soc
PubMed ID27564673
PubMed Central IDPMC5059492
Grant ListU01 HL066952 / HL / NHLBI NIH HHS / United States