Gene therapy progress and prospects: Cystic fibrosis

被引：61

作者：

Griesenbach, U ^{[1
]}

Ferrari, S ^{[1
]}

Geddes, DM ^{[1
]}

Alton, EWFW ^{[1
]}

机构：

[1] Univ London Imperial Coll Sci Technol & Med, Dept Gene Therapy, Fac Med, Natl Heart & Lung Inst, London SW3 6LR, England

来源：

GENE THERAPY | 2002年 / 9卷 / 20期

关键词：

cystic fibrosis; gene therapy; airway gene transfer;

D O I：

10.1038/sj.gt.3301791

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

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：7

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