Non-Technical Abstract 
2. Non-Technical Abstract. Cystic fibrosis (CF) is the most common lethal 
hereditary disorder of Caucasians in the U.S. The major manifestations are in 
the lung, with thick, sticky mucus, chronic infections and inflammation. The 
average life span is approximately 28 years. The disease is caused by inherit- 
ed abnormalities of the cystic fibrosis transmembrane conductance regulator 
(CFTR) gene. Because of these genetic abnormalities, the cells lining the air- 
ways cannot move salt in a normal fashion, causing the mucus abnormalities, 
infection and inflammation and consequent progressive damage to the lung. In 
the context of laboratory studies demonstrating that transfer of the normal 
CFTR gene to the airway cells of individuals with CF will compensate for the 
abnormalities in the CFTR gene and permit airway cells to function in a normal 
fashion, this protocol is directed toward using a modified adenovirus to trans- 
fer the normal CFTR gene into the cells lining the airways of individuals with 
CF. Following a period of baseline evaluation, a defective adenovirus (which 
reduces the possibility of multiplication) will be instilled into the airways 
of individuals with CF. A variety of biologic and clinical parameters will be 
used to assess safety and biologic efficacy (the ability of the treatment to 
correct the abnormalities in the cells lining of the airways as assessed by 
laboratory tests). A total of 26 patients will receive the modified adenovi- 
rus, at various doses and time intervals. At the conclusion of this study, it 
should be possible to assess whether this strategy of compensating for the 
genetic abnormalities of CF will be a rational approach to treat the respi- 
ratory manifestations of this disease on a continuing basis. 
Recombinant DNA Research, Volume 20 
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