Abstract
This chapter discusses the utility of the adenoviral vectors in animal models of genetic diseases. It is mainly focused on the therapeutic uses of adenoviral vectors in cystic fibrosis (CF) lung disease. CF lung disease has been considered as a prototypic disease state for "proof-of-concept" gene-therapy strategies. The cloning of the gene responsible for CF pathophysiology, two groundbreaking observations made gene therapy for CF lung disease appear imminent. First, isolated epithelial cells cultured from the airway epithelium of CF patients could be phenotypically "corrected" by transferring into the cells the cDNA corresponding to the CF gene. Second, adenoviral (Ad) vectors engineered to express the CF gene were administered to the airways of experimental animals and transgene expression observed in cells that were considered to require "correction." These findings will not only be beneficial to the treatment of CF lung disease but also other disease states, which are continually being brought closer to a treatment and perhaps a cure by this new and exciting biomedical technology.
Published Version
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