4.5 Review

Gene therapy progress and prospects: cystic fibrosis

期刊

GENE THERAPY
卷 13, 期 14, 页码 1061-1067

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NATURE PUBLISHING GROUP
DOI: 10.1038/sj.gt.3302809

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cystic fibrosis; airway gene transfer; viral vectors; non-viral vectors

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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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