Abstract
The first safe and effective partial correction of the genetic defect in cystic fibrosis (CF) has been recently reported by researchers from the UK. CF is caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which encodes a cAMP-regulated chloride channel. If the gene is defective, chloride transport on the apical surface of epithelial cells is impaired and absorption of sodium ions increases. By transferring a normal CFTR gene to the lungs and noses of CF patients, Eric Alton (Imperial College, London, UK) and colleagues have partially corrected chloride abnormalities in these tissues and have reduced bacterial adherence to CF lung tissue 1 Alton E.W.F. et al. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled study. Lancet. 1999; 353: 947-954 Abstract Full Text Full Text PDF PubMed Scopus (374) Google Scholar .
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