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Basic science for the chest physician
Delivery of genes into the CF airway
  1. Deborah R Gill1,2,
  2. Stephen C Hyde1,2
  1. 1Gene Medicine Research Group, Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, John Radcliffe Hospital, Oxford, UK
  2. 2UK Cystic Fibrosis Gene Therapy Consortium, Imperial College London, University of Edinburgh, University of Oxford, Oxford, UK
  1. Correspondence to Dr Stephen C Hyde, Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, John Radcliffe Hospital, University of Oxford, Oxford OX3 9DU, UK; steve.hyde{at}ndcls.ox.ac.uk

Abstract

Gene therapy was suggested as a potential treatment for cystic fibrosis (CF), even before the identification of the CFTR gene. Initial enthusiasm has been tempered as it became apparent that reintroduction of the CFTR gene into the cells of the lung is more difficult than anticipated. Here, we review the major gene delivery vectors evaluated clinically, and suggest that advances in either plasmid DNA design and/or hybrid lentivirus biology may finally facilitate lung gene transfer with efficiencies sufficient for CF gene therapy to offer clinical benefit.

  • Cystic Fibrosis

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