Am. J. Respir. Cell Mol. Biol., Vol 14, No. 1, Jan 1996, 1-18.
Gene therapy approaches for inherited and acquired lung diseases
DT Curiel, JM Pilewski and SM Albelda
Gene Therapy Program, UAB Comprehensive Cancer Center, University of Alabama at Birmingham, USA.
Gene therapy is the treatment of any disorder or pathophysiologic state
based upon the transfer of genetic information. The lung represents a major
target of gene therapy for the treatment of genetic disorders such as
cystic fibrosis and alpha 1-antitrypsin deficiency. Other diseases are also
being targeted, including pulmonary inflammation, surfactant deficiency,
pulmonary hypertension, lung cancer, and malignant mesothelioma. This
review will examine some general concepts regarding gene transfer and gene
therapy, provide an overview of the current vectors being developed to
achieve safe and efficient gene transfer, and summarize the ongoing work to
apply this new technology to the treatment of both inherited and acquired
pulmonary diseases. Although tremendous progress has been made in the
ability to successfully transfer genes to cells, there are several
unresolved problems limiting the clinical application of this technology to
human pulmonary disease. However, as vector technology evolves, gene
therapy may become a reality for a number of lung diseases.
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Copyright © 1996 American Thoracic Society.
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