1
views
0
recommends
+1 Recommend
0 collections
    0
    shares
      • Record: found
      • Abstract: found
      • Article: not found

      Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease.

      Read this article at

      ScienceOpenPublisherPMC
      Bookmark
          There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.

          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.

          Related collections

          Author and article information

          Journal
          Ann Am Thorac Soc
          Annals of the American Thoracic Society
          American Thoracic Society
          2325-6621
          2325-6621
          Aug 2016
          : 13 Suppl 4
          Affiliations
          [1 ] Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.
          Article
          10.1513/AnnalsATS.201506-344KV
          5059492
          27564673

          Comments

          Comment on this article