Gene Therapy for CF using Cationic Liposome Mediated Gene Transfer: Phase I Trial 
in clinical status of patients involved in this study, participants in this trial will not benefit 
directly. However, because the nasal airway functions similarly to lower airways, and exhibits 
the CF transport defect, safe, gene-transfer mediated correction of the ion transport defect within 
the nasal airway would establish the feasibility of a trial of lipid mediated gene transfer within 
the lungs of CF patients. Because lung disease represents the predominant cause of death in 
patients with CF, the information gathered in this study is therefore very likely to benefit the 
CF community as a whole. Most patients with CF die before age 30 despite the best available 
therapies. Adenovirus-based delivery of CFTR is still in the experimental stages and little is 
known either about safety or efficacy in human trials. Since five independent laboratories have 
now reported safe and efficient gene transfer of CFTR or other reporter genes to non-human, 
mammalian respiratory epithelium using cationic liposomes, it is reasonable to evaluate this 
potential avenue for gene delivery in a limited Phase I trial in humans. The information gained 
from this study will add to our understanding of lipid based gene delivery and will serve the 
critical need of developing new and better therapies for CF lung disease. 
Recombinant DNA Research, Volume 18 
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