Gene Therapy for Lung Disease: Hype or Hope?

Gene Therapy for Lung Disease: Hype or Hope?

From University of Pennsylvania Medical Center, Philadelphia, Pennsylvania.

Abstract

Gene therapy, the treatment of any disorder or pathophysiologic 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 α₁-antitrypsin deficiency, and for other diseases, including lung cancer, malignant mesothelioma, pulmonary inflammation, surfactant deficiency, and pulmonary hypertension. This paper examines general concepts 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 advancements 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 important tool for the 21st-century clinician.

Article and Author Information

For definitions of terms, see Glossary at end of text.
Acknowledgments: The authors thank Kate Darwin and Linda Kalb for assistance in preparing the manuscript.
Grant Support: This work was supported by grant PO1 66726 from the National Cancer Institute, National Institutes of Health; the Benjamin Shein Foundation for Humanity; and the Lunenfield Foundation (Dr. Albelda). Dr. Wiewrodt is a postdoctoral fellow of Mildred Scheel Stiftung für Krebsforschung der Deutschen Krebshilfe e.V. (grant D/98/02288).
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Current Author Addresses: Dr. Albelda: 856 BRBII/III, 421 Curie Boulevard, Philadelphia, PA 19104.
Dr. Wiewrodt: 835 BRBII/III, 421 Curie Boulevard, Philadelphia, PA 19104.
Dr. Zuckerman: Pulmonary and Critical Care, Maine Medical Center, 22 Bramhall Street, Portland, ME 04102.