Cystic fibrosis pulmonary gene therapy

Pulmonary gene therapy is attractive for the treatmment of chronic bronchitis, cystic fibrosis, a-1 antitrypsin deficiency, familial emphysema, asthma, pulmonary infections, surfactant deficiency, pulmonary hypertension, lung cancer, and malignant mesothelioma. The pulmonary endothelium may act as a bioreactor for the production and secretion of therapeutic proteins, such as clotting factors and erythropoietin into the blood circulation. There is a potential benefit for acquired lung diseases, as well as cancers, to be controlled and possibly treated by expression of cytokines, surfactant, antioxidant enzymes, or mucoproteins within lung cells. 

Addresses nev. imaging research appivaches relevant to studies of pulmonary gene therapy, cystic fibrosis, lung cancer, asthma, and acute lung injury, among others. 

Pulmonary delivery of liposomes has focused on the treatment of asthma, infectious diseases, genetic diseases (cystic fibrosis), and lung injury and lately on gene therapy. 

Although aerosols of various forms for treatment of respiratory disorders have been in use since the middle of the twentieth century, the interest in the use of pulmonary route for systemic drug delivery is recent. Interest in this approach has been further stimulated by the demonstration of potential utility of lung as a portal for entry of peptides and the feasibility of gene therapy for cystic fibrosis. It is important to understand the mechanism of macromolecule absorption by the lungs for an effective use of this route. 

Since the early experimental success with gene therapy in the 1990s, the clinical use of gene therapy has been proven to be difficult. Phase I clinical trials for pulmonary diseases have focused primarily on cystic fibrosis and more recently on lung cancer (109,110). Although gene therapy for lung transplantation has seen increased experimental success, no clinical trials have been conducted. 

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