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Adeno-associated Virus and Lentivirus Pseudotypes for Lung-directed Gene Therapy

Gene Therapy Program, Division of Medical Genetics, Department of Medicine, University of Pennsylvania Health System, and The Wistar Institute, Philadelphia, Pennsylvania

Correspondence and requests for reprints should be addressed to James M. Wilson, M.D., Ph.D., 204 Wistar Institute, 3601 Spruce Street, Philadelphia, PA 19104-4268. E-mail: wilsonjm{at}mail.med.upenn.edu

The enthusiasm for cystic fibrosis gene therapy that attended the initial cloning of the gene and in vitro correction of the genetic defect eventually diminished as we learned more about the limitations of vector technologies that were available in the 1980s and 1990s. Substantial progress has been made, however, over the last 5 years in developing second- and third-generation vector constructs that should be more useful in achieving gene transfer to the lung for the treatment of pulmonary diseases such as cystic fibrosis.

Key Words: recombinant • AAV • lentivirus • lung tropism

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