Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

• Published in: Nature medicine Vol. 1; no. 1; pp. 39 - 46
• Main Authors: Caplen, Natasha J., Alton, Eric W.F.W., Mddleton, Peter G., Dorin, Julia R., Stevenson, Barbara J., Gao, Xiang, Durham, Stephen R., Jeffery, Peter K., Hodson, Margaret E., Coutelle, Charles, Huang, Leaf, Porteous, David J., Williamson, Robert, Geddes, Duncan M.
• Format: Journal Article
• Language: English
• Published: New York Nature Publishing Group US 01.01.1995; Nature Publishing Group
• Subjects: Base Sequence; Biomedical and Life Sciences; Biomedicine; Cancer Research; Chlorides - metabolism; Clinical trials; Complementary DNA; Conductance; Cystic fibrosis; Cystic Fibrosis - therapy; Cystic fibrosis transmembrane conductance regulator; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Deoxyribonucleic acid; DNA; DNA Primers - chemistry; DNA, Complementary; Double-Blind Method; Epithelium; Gene Expression; Gene transfer; Gene Transfer Techniques; Genetic Therapy - methods; Humans; Infectious Diseases; Liposomes; Male; Membrane Potentials; Metabolic Diseases; Molecular Medicine; Molecular Sequence Data; Nasal Mucosa; Neurosciences; Perfusion; Plasmids; Restoration; Ribonucleic acid; RNA; RNA, Messenger - genetics; Transfection; Transgenes

We report the results of a double-blind, placebo-controlled trial in nine cystic fibrosis (CF) subjects receiving cationic liposome complexed with a complementary DNA encoding the CF transmembrane conductance regulator (CFTR), and six CF subjects receiving only liposome to the nasal epithelium. No a...