Gene therapy for lung disease: hype or hope

Gene therapy, the treatment of any disorder or pathophys-iologic state on the basis of the transfer of genetic infor-mation, 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 a1-antitrypsin deficiency, and for other diseases, including lung cancer, malignant mesothelioma, pulmonary inflammation, surfactant deficiency, and pul-monary hypertension. This paper examines general con-cepts in gene therapy, summarizes the results of published clinical trials, and highlights areas of research aimed at overcoming challenges in the field. Although progress has been slower than anticipated, gene transfer has been safely achieved in patients with lung diseases. Recent ad-vancements in understanding of the molecular basis of lung disease and the development of improved vector systems make it likely that gene therapy will be an impor-tant tool for the 21st-century clinician. Ann Intern Med. 2000;132:649-660