Format

Send to

Choose Destination
  • Filters activated: Field: Title Word. Clear all
Ann Am Thorac Soc. 2016 Aug;13 Suppl 4:S352-69. doi: 10.1513/AnnalsATS.201506-344KV.

Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease.

Author information

1
Department of Genetic Medicine, Weill Cornell Medical College, New York, New York.

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.

KEYWORDS:

adeno-associated vector; adenovirus vectors; alpha-1 antytripsin deficiency; gene therapy; lung disease

PMID:
27564673
PMCID:
PMC5059492
DOI:
10.1513/AnnalsATS.201506-344KV
[Indexed for MEDLINE]
Free PMC Article

Supplemental Content

Full text links

Icon for Atypon Icon for PubMed Central
Loading ...
Support Center