Hum Mol Genet. 2013 Oct 15;22(R1):R52-8. doi: 10.1093/hmg/ddt372. Epub 2013 Aug 4.
Since cloning of the CFTR gene more than 20 years ago a large number of pre-clinical and clinical CF gene therapy studies have been performed and a vast amount of information and know-how has been generated. Here, we will review key studies with a particular emphasis on clinical findings. We have learnt that the lung is a more difficult target than originally anticipated, and we describe the strength and weaknesses of the most commonly used airway gene transfer agents (GTAs). In our view, one of the most significant developments in recent years is the generation of lentiviral vectors, which efficiently transduce lung tissue. However, focused and co-ordinated efforts assessing lentiviral vector safety and scaling up of production will be required to move this vector into clinical lung gene therapy studies.
自 20 多年前 CFTR 基因克隆以来，已经进行了大量的临床前和临床 CF 基因治疗研究，并产生了大量的信息和技术诀窍。在这里，我们将重点回顾关键研究，特别是临床发现。我们已经了解到，肺部比最初预期的更难成为治疗靶点，我们还描述了最常用的气道基因转移剂（GTAs）的优缺点。在我们看来，近年来最重要的发展之一是产生了能够有效转导肺部组织的慢病毒载体。然而，需要集中协调的努力来评估慢病毒载体的安全性并扩大生产规模，以将这种载体应用于临床肺部基因治疗研究。
