Taking stock of gene therapy for cystic fibrosis

Respiratory Research

Eric WFW Alton¹  Duncan M Geddes¹  Myra Stern¹ 

[1]Imperial College at the National Heart and Lung Institute, London, UK

关键词: recombinant viruses;    gene therapy;    cystic fibrosis;    CFTR;    cationic lipids;   

Others  :  1227483
DOI  :  10.1186/rr16

 received in 2000-04-28, accepted in 2000-09-01,  发布年份 2000

【 摘 要 】

The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal models in vivo has been followed by numerous clinical studies using both viral and non-viral vectors to transfer normal copies of the gene to the lungs and noses of CF patients. A wealth of data have emerged from these studies, reflecting enormous progress and also helping to focus and define key difficulties that remain unresolved. Gene therapy for CF remains the most promising possibility for curative rather than symptomatic therapy.

【 授权许可】

   
2000 Current Science Ltd

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