Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial.

Background: We and others have previously reported significant changes in chloride transport after cationic-lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the nasal epithelium of patients with cystic fibrosis. We studied the safety and efficacy of this gene transfer to the lungs and nose of patients with cystic fibrosis in a double-blind placebo-controlled trial.

Methods: Eight patients with cystic fibrosis were randomly assigned DNA-lipid complex (active) by nebulisation into the lungs followed 1 week later by administration to the nose.

The damaging role of bacteria in chronic lung infection.

Because of their embryological derivation from foregut the respiratory airways are regularly host to bacteria arriving in inspired air and aspirated from the naso-oropharynx. Should the first-line defence (mucociliary clearance) be less than perfect, such bacteria remain longer in the airway mucus and some are able to capitalize on this delay by compromising mucus transport further through the production of epithelium-damaging cilioinhibitory and ciliotoxic substances. Bacterial persistence in the airways elicits an inflammatory host response which causes further damage to the airway and results in a 'vicious circle' of progressive lung damage.