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Non–invasive liposome–mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice

Nature Genetics volume 5, pages 135–142 (1993)Cite this article

A Correction to this article was published on 01 November 1993

Abstract

We report gene transfer to the Edinburgh insertional mutant mouse (cf/cf), delivering CFTR cDNA–liposome complexes into the airways by nebulization. We show full restoration of cAMP related chloride responses in some animals and demonstrate, in the same tissues, human CFTR cDNA expression. Overall, a range of correction was seen with restoration of about 50% of the deficit between wild type mice and untreated cf/cf controls. We report modest correction in the intestinal tract following direct instillation and provide initial encouraging safety data for both the respiratory and intestinal tract following the liposome mediated gene delivery. The non–viral nature and potentially lower immunogenicity of DNA–liposomes suggest that this may offer a therapeutic alternative to adenoviral therapies.

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Authors and Affiliations

  1. Ion Transport Laboratory, National Heart and Lung Institute, London, SW3 6LR, UK

    E. W. F. W. Alton, P. G. Middleton, S. N. Smith, D. M. Steel, F. M. Munkonge & D. M. Geddes

  2. Department of Biochemistry and Molecular Genetics, St Mary's Hospital Medical School, London, W2 1PG, UK

    N. J. Caplen, S. L. Hart & R. Williamson

  3. Department of Lung Pathology, National Heart and Lung Institute, London, SW3 6LR, UK

    P. K. Jeffery

  4. Department of Chemistry, Imperial College, London, SW7 1NA, UK

    K. I. Fasold & A. D. Miller

  5. MRC Human Genetics Unit, Western General Hospital, Edinburgh, EH4 2XU, UK

    P. Dickinson, B. J. Stevenson, G. McLachlan, J. R. Dorin & D. J. Porteous

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  1. E. W. F. W. Alton
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Alton, E., Middleton, P., Caplen, N. et al. Non–invasive liposome–mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat Genet 5, 135–142 (1993). https://doi.org/10.1038/ng1093-135

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