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Gene therapy progress and prospects: cystic fibrosis

Abstract

Our first review on progress and prospects in cystic fibrosis (CF) gene therapy was published in this series in October 2002. We now summarize the progress made since then and comment on the prospects for CF gene therapy over the next couple of years. Three clinical trials have been carried out, further supporting the proof-of-principle that gene transfer to the airway epithelium is feasible. Developments in viral and non-viral vectors, as well as recent alternative strategies such as gene repair, trans-splicing and stem cell therapy will be reviewed.

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Acknowledgements

We thank all members of the UK CF Gene Therapy Consortium Strategy Group (Chris Boyd, Jane Davies, Deborah Gill, Steve Hyde and David Porteous) for critical reading and thank Lucinda Somerton for help with preparation of the manuscript.

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Correspondence to U Griesenbach.

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Griesenbach, U., Geddes, D., Alton, E. et al. Gene therapy progress and prospects: cystic fibrosis. Gene Ther 13, 1061–1067 (2006). https://doi.org/10.1038/sj.gt.3302809

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