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The efficacy of the in vivo transfer of complementary DNA is evaluated by the use of mice having the disrupted cystic fibrosis (CF) gene. The CF defect is corrected partially in Cl ion transport in vivo even after repeated administration of high doses of adenovirus vectors. The adenovirus vectors are not capable of correcting the sodium ion transport defect. A comparison of human and mice CF genes indicates that a difference in susceptibility to the adenovirus-5 transduction exists in human and mice genes.

Discusses the efficacy of complementary DNA transfer which encodes cystic fibrosis transmembrane regulator to the affected columnar epithelial cells lining the lung airways. Pre-clinical studies in vitro of the transfer system; Efficacy of the system in vivo; Partial correction of the cystic fibrosis defect; Discrepancy of the in vivo and in vitro transfer efficiency.