Gene Therapy Progress and Prospects: Cystic fibrosis

被引：0
作者：
U Griesenbach
S Ferrari
D M Geddes
E W F W Alton
机构：
[1] National Heart and Lung Institute,Department of Gene Therapy
[2] Imperial College,undefined
[3] Faculty of Medicine,undefined
来源：
Gene Therapy | 2002年 / 9卷
关键词：
cystic fibrosis; gene therapy; airway gene transfer;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
Since the cloning of the cystic fibrosis gene (CFTR) in 1989, 18 clinical trials have been carried out, including five in the 2 years reviewed here. Most trials demonstrated proof-of-principle for gene transfer to the airway. However, gene transfer efficiency with each of the three gene transfer agents (adenovirus (Ad), adeno-associated virus 2 (AAV2) and cationic liposomes) was low, and most likely insufficient to achieve clinical benefit. Here, we will review the clinical and pre-clinical progress for the last 2 years (2000–2001) and briefly speculate on future prospects for the next 2 in CF gene therapy.
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页码：1344 / 1350
页数：6
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