Methods for the Use of Retroviral Vectors for Transfer of the CFTR Gene to Airway Epithelium.
Cystic fibrosis (CF) is a recessive genetic disease that affects the regulation of ion transport in the epithelia of various organs in the body including the lungs, pancreas, intestine, salivary glands, and urogenital tract. The protein encoded by the CF gene is an integral plasma membrane protein called the cystic fibrosis transmembrane conductance regulator (CFTR) and has been shown to function as a chloride channel (1). In the lungs, CFTR dysfunction affects electrolyte and fluid transport across the apical membrane of airway epithelial cells. There, sodium hyperabsorption and defective chloride secretion lead to dehydration of the fluids on the airway surface and, in turn, this leads to chronic infections and severe damage. The severity of CF lung disease and the potential accessibility of the airways to gene transfer vectors has led to proposals that gene therapy be applied for the treatment of CF lung disease (2).
Cystic Fibrosis/Pulmonary Research and Treatment Center, Department of Medicine, University of North Carolina, Chapel Hill, NC.
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Olsen
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Johnson
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Yankaskas