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

摘要: Summary 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 nasal epithelium patients with fibrosis. studied safety efficacy this lungs nose a double-blind placebo-controlled trial. Methods Eight were randomly assigned DNA-lipid complex (active) by nebulisation into followed 1 week later administration nose. control same protocol but lipid alone (placebo). Safety was assessed clinically, radiography, pulmonary function, induced sputum, histological analysis. Efficacy analysis vector-specific DNA mRNA, in-vivo potential difference, epifluorescence assay efflux, bacterial adherence. Findings Seven eight receiving active mild influenza-like symptoms that resolved within 36 h. Six both placebo groups airway over period 12 h following administration. No specific treatment required for either event. Pulmonary resulted (p Interpretation Cationic-lipid-mediated can significantly influence underlying defect