ABSTRACT
Gene therapy has potential as a future medical treatment for chronic inflammatory lung diseases. Although the genetic bases for these diseases are different, both disorders are characterized by a chronic, neutrophil-dominated, inflammatory lung process. The most common genetic abnormality associated with premature emphysema is the Zallele. Unopposed elastolytic destruction of the lung parenchyma and subsequent panacinar emphysema ensues. Neutrophils are recruited into the lungs by various stimuli and release elastase and other potentially toxic substances. The liver-directed gene therapy models previously described demonstrated successful gene transfer and persistent gene expression. Clinical manifestations of the disease occur primarily in the lung, liver, pancreas, and intestinal tract. Males are usually sterile. Chronic inflammation with large numbers of activated neutrophils in the lungs can cause pulmonary damage due to the release of neutrophil elastase and other potentially toxic substances.
