Davies J C, Geddes D M, Alton E W
Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London, UK.
J Gene Med. 2001 Sep-Oct;3(5):409-17. doi: 10.1002/jgm.200.
Cystic fibrosis (CF) is associated with significant morbidity and mortality, despite significant advances in conventional treatment. The field of gene therapy has progressed rapidly since the cystic fibrosis transmembrane conductance regulator (CFTR) gene was cloned. In this review we discuss current knowledge on the underlying molecular defect in CF, and the progress in gene transfer studies from the early in vitro work through to clinical trials, including the development of endpoints to assess efficacy. We highlight the problems encountered, and likely future directions of the field.
尽管传统治疗取得了显著进展，但囊性纤维化（CF）仍与严重的发病率和死亡率相关。自囊性纤维化跨膜传导调节因子（CFTR）基因被克隆以来，基因治疗领域发展迅速。在本综述中，我们讨论了目前关于CF潜在分子缺陷的知识，以及从早期体外研究到临床试验的基因转移研究进展，包括评估疗效的终点指标的发展。我们强调了遇到的问题以及该领域可能的未来发展方向。
