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Delivery of genes into the CF airway

Deborah R Gill¹, Stephen C Hyde¹

¹Gene Medicine Research Group, Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, John Radcliffe Hospital, Oxford, UK UK Cystic Fibrosis Gene Therapy Consortium, Imperial College London, University of Edinburgh, University of Oxford, Oxford, UK.

|July 13, 2014

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.

Keywords:

Cystic Fibrosis

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