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

N. J. Caplen, E. W. Alton, P. G. Middleton, J. R. Dorin, B. J. Stevenson, X. Gao, S. R. Durham, P. K. Jeffery, M. E. Hodson, C. Coutelle, L. Huang, D. Porteous, R. Williamson, D. Geddes

Research output: Contribution to journalArticlepeer-review

Abstract / Description of output

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 adverse clinical effects were seen and nasal biopsies showed no histological or immuno-histological changes. A partial restoration of the deficit between CF and non-CF subjects of 20% was seen for the response to low Cl- perfusion following CFTR cDNA administration. This was maximal around day three and had reverted to pretreatment values by day seven. In some cases the response to low Cl- was within the range for non-CF subjects. Plasmid DNA and transgene-derived RNA were detected in the majority of treated subjects. Although these data are encouraging, it is likely that transfection efficiency and the duration of expression will need to be increased for therapeutic benefit.
Original languageEnglish
Pages (from-to)39-46
Number of pages8
JournalNature Medicine
Volume1
Issue number1
Publication statusPublished - 1995

Keywords / Materials (for Non-textual outputs)

  • Base Sequence Chlorides/metabolism Cystic Fibrosis/*therapy Cystic Fibrosis Transmembrane Conductance Regulator/*genetics DNA Primers/chemistry DNA, Complementary Double-Blind Method Gene Expression Gene Therapy/*methods *Gene Transfer Techniques Humans Liposomes Male Membrane Potentials Molecular Sequence Data Nasal Mucosa RNA, Messenger/genetics Transgenes

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