Gene therapy for cystic fibrosis: an example for lung gene therapy
Gene therapy for cystic fibrosis: an example for lung gene therapy
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DOI：
10.1038/sj.gt.3302368
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发表时间：
2004-10-01
期刊：
影响因子：
5.1
通讯作者：
Alton, EWFW
中科院分区：
文献类型：
--
作者：
Griesenbach, U;Geddes, DM;Alton, EWFW
目前正在评估基因治疗用于各种急性和慢性肺部疾病。将基因转移到复杂肺结构的单个细胞类型中的要求将在很大程度上取决于靶疾病。在过去的十年中，基因治疗界已经认识到，甚至没有一种载体适用于所有应用，但基因转移剂必须仔细选择。基因治疗对于目前没有令人满意的治疗选择的疾病特别有吸引力，并且对于单基因疾病可能比复杂疾病更容易。囊性纤维化（CF）符合这些标准，因此是基于基因治疗的治疗的良好候选者。本文将重点介绍CF作为肺基因治疗的一个例子，并讨论在这一领域取得的进展，在过去的几年里。
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.