Title | |
Publication Type | Journal Article |
Year of Publication | 2016 |
Authors | Chiuchiolo MJ, Crystal RG |
Journal | Ann Am Thorac Soc |
Volume | 13 Suppl 4 |
Pagination | S352-69 |
Date Published | 2016 Aug |
ISSN | 2325-6621 |
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 |
Alternate Journal | Ann Am Thorac Soc |
PubMed ID | 27564673 |
PubMed Central ID | PMC5059492 |
Grant List | U01 HL066952 / HL / NHLBI NIH HHS / United States |