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Gene Therapy for Lung Disease: Hype or Hope?

Steven M. Albelda, MD; Rainer Wiewrodt, MD; and Jonathan B. Zuckerman, MD
[+] Article and Author Information

From University of Pennsylvania Medical Center, Philadelphia, Pennsylvania.


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).

Requests for Single Reprints: Steven M. Albelda, MD, 856 BRBII/III, 421 Curie Boulevard, Philadelphia, PA 19104; e-mail, albelda@mail.med.upenn.edu.

Requests To Purchase Bulk Reprints (minimum, 100 copies): the Reprints Coordinator; phone, 215-351-2657; e-mail, reprints@mail.acponline.org.

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.


Ann Intern Med. 2000;132(8):649-660. doi:10.7326/0003-4819-132-8-200004180-00008
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Approximately 10 years ago, advances in molecular genetics and gene transfer technology made possible the development of gene therapy, modification of the genetic makeup of cells for therapeutic purposes. Although gene therapy was originally proposed for treatment of inherited recessive disorders in which transfer of a normal copy of a single defective gene might prevent the development of disease or slow its progression (1), it soon became clear that reasonable targets for gene therapy extended beyond traditional genetic disorders into the realm of acquired diseases, such as vascular diseases and cancer. The concept of gene therapy now encompasses the treatment of any pathophysiologic state on the basis of the transfer of genetic material, including complementary DNA, full-length genes, RNA, or oligonucleotides.

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