Alpha 1-antitrypsin deficiency is a genetically inherited condition that affects the lungs and/or the liver. It is passed on from parents to their children through genes. It is a progressive condition, which means it may worsen over time. AAT normally protects the lung from damage. In this condition, the AAT protein is abnormal and cannot be released from the liver to protect the lungs. This can result in chronic obstructive pulmonary disease (COPD). It can lead to serious lung and/or liver disease.

For the current therapy, symptoms can be treated with standard medical therapy (such as bronchodilators), augmentation therapy (giving patients the AAT protein by intravenous infusion, usually every week or every month) and/or behavioral measures (such as smoking cessation).
   
This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for the AAT protein, which if safe and efficacious, and will protect the lung on a persistent basis. The term, ‘gene therapy’ is used to refer to the process of transferring a missing gene through a vector to reduce symptoms of disease.
   
In this study, we hope to learn the safety/toxicity and initial evidence of efficacy of intravenous delivery of gene therapy to AAT deficient individuals.