Gene therapy for lung disease: Hype or hope?

Gene therapy, the treatment of any disorder or pathphysiologic state on the basis of the transfer of genetic information, was a high-priority goal in the 1990s. The lung is a major target of gene therapy for genetic disorders , such as cystic fibrosis and alpha(1)-antitrypsin deficiency, and for othe r diseases, including lung cancer, malignant mesothelioma, pulmonary inflam mation, surfactant deficiency, and pulmonary hypertension. This paper exami nes general concepts in gene therapy, summarizes the results of published c linical trials, and highlights areas of research aimed at overcoming challe nges in the field. Although progress has been slower than anticipated, gene transfer has been safely achieved in patients with lung diseases. Recent a dvancements in understanding of the molecular basis of lung disease and the development of improved vector systems make it likely that gene therapy wi ll be an important tool for the 21st-century clinician.