Alton E W, Geddes D M
Royal Brompton Hospital, National Heart and Lung Institute, London, UK.
Gene Ther. 1995 Mar;2(2):88-95.
Since the isolation of the cystic fibrosis (CF) gene in 1989, the potential for gene therapy for this disease has existed. Although current treatments have resulted in a mean life expectancy of approximately 30 years, there is clearly a need for more effective therapy. A large number of studies have now assessed both in vitro and in vivo CFTR gene transfer into cell lines, animal models and most recently in CF subjects. Despite this rapid progress several difficulties remain including efficient in vivo gene transfer and the measurement of end-points to assess such gene transfer. This article reviews some of the clinically related aspects of gene therapy for CF.
自1989年囊性纤维化（CF）基因被分离出来后，针对这种疾病进行基因治疗的可能性就已存在。尽管目前的治疗方法已使平均预期寿命达到了约30岁，但显然仍需要更有效的治疗方法。现在已有大量研究评估了在体外和体内将囊性纤维化跨膜传导调节因子（CFTR）基因转移到细胞系、动物模型以及最近在CF患者体内的情况。尽管取得了如此迅速的进展，但仍存在一些困难，包括有效的体内基因转移以及评估此类基因转移的终点指标的测定。本文综述了CF基因治疗中一些与临床相关的方面。
