Ontology highlight
ABSTRACT:
SUBMITTER: Chiuchiolo MJ
PROVIDER: S-EPMC5059492 | biostudies-literature | 2016 Aug
REPOSITORIES: biostudies-literature
Chiuchiolo Maria J MJ Crystal Ronald G RG
Annals of the American Thoracic Society 20160801
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 t ...[more]