Gene Medicine Research Group, Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, John Radcliffe Hospital, Oxford, UK UK Cystic Fibrosis Gene Therapy Consortium, Imperial College London, University of Edinburgh, University of Oxford, Oxford, UK.
Thorax. 2014 Oct;69(10):962-4. doi: 10.1136/thoraxjnl-2014-205835. Epub 2014 Jul 11.
Gene therapy was suggested as a potential treatment for cystic fibrosis (CF), even before the identification of the CFTR gene. Initial enthusiasm has been tempered as it became apparent that reintroduction of the CFTR gene into the cells of the lung is more difficult than anticipated. Here, we review the major gene delivery vectors evaluated clinically, and suggest that advances in either plasmid DNA design and/or hybrid lentivirus biology may finally facilitate lung gene transfer with efficiencies sufficient for CF gene therapy to offer clinical benefit.
基因治疗被认为是囊性纤维化（CF）的一种潜在治疗方法，甚至在 CFTR 基因被发现之前就是如此。随着 CFTR 基因重新导入肺部细胞的难度超出预期变得明显，最初的热情有所减退。在这里，我们回顾了临床上评估的主要基因传递载体，并提出质粒 DNA 设计和/或杂交慢病毒生物学方面的进展最终可能会促进肺基因转移，效率足以使 CF 基因治疗为患者带来临床获益。
