Griesenbach U, Geddes D M, Alton E W F W
Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK.
Gene Ther. 2004 Oct;11 Suppl 1(Suppl 1):S43-50. doi: 10.1038/sj.gt.3302368.
Gene therapy is currently being evaluated for a wide range of acute and chronic lung diseases. The requirement of gene transfer into the individual cell types of the complex lung structure will very much depend on the target disease. Over the last decade, the gene therapy community has recognized that there is not even one vector that is good for all applications, but that the gene transfer agent has to be carefully chosen. Gene therapy is particularly attractive for diseases that currently do not have satisfactory treatment options and probably easier for monogenic disorders than for complex diseases. Cystic fibrosis (CF) fulfills these criteria and is therefore a good candidate for gene therapy-based treatment. This review will focus on CF as an example for lung gene therapy and discuss the progress made in this field over the last couple of years.
目前正在对多种急慢性肺部疾病进行基因治疗评估。将基因导入复杂肺部结构的各个细胞类型的需求在很大程度上取决于目标疾病。在过去十年中,基因治疗领域已经认识到,没有一种载体适用于所有应用,而是必须仔细选择基因传递剂。基因治疗对于目前没有令人满意治疗选择的疾病特别有吸引力,而且对于单基因疾病可能比对复杂疾病更容易。囊性纤维化(CF)符合这些标准,因此是基于基因治疗的良好候选对象。本综述将以CF为例探讨肺部基因治疗,并讨论过去几年该领域取得的进展。