A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis

摘要: Cystic fibrosis (CF) is a common, serious, inherited dis- Biopsies of the nasal epithelium taken 7 days after dosing ease. The major cause mortality in CF lung disease, were normal. No significant changes clinical parameters due to failure airway epithelial cells express observed. Functional expression CFTR assessed functional product cystic transmembrane by vivo potential difference measurements showed conductance regulator (CFTR) gene. A treatment transient correction chloride transport abnorfor disease air- mality two patients (15 one patient). ways following gene transfer. We have undertaken Fluorescence microscopy demonstrated function ex double-blinded, placebo-controlled, study from brushings. In total, evidence transfer cDNA 12 was obtained six out patients. Cationic liposomes complexed with plasmid eight treated These results provide proof containing human administered concept for liposome-mediated patients, whilst four received placebo.