Review
Gene Therapy (2006) 13, 1061–1067. doi:10.1038/sj.gt.3302809
Gene therapy progress and prospects: cystic fibrosis
U Griesenbach1,2, D M Geddes1,2 and E W F W Alton1,2 on behalf of the UK Cystic Fibrosis Gene Therapy Consortium
- 1Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London, UK
- 2The UK Cystic Fibrosis Gene Therapy Consortium, London, UK
Correspondence: Dr U Griesenbach, Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW3 6LR, UK. E-mail: u.griesenbach@imperial.ac.uk
Received 13 January 2006; Revised 2 May 2006; Accepted 5 May 2006.
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.
Keywords:
cystic fibrosis, airway gene transfer, viral vectors, non-viral vectors
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