Department of Pediatrics, Ludwig-Maximilians-University Munich, Lindwurmstrasse 2a, 80337 Munich, Germany.
Int J Pharm. 2010 May 5;390(1):84-8. doi: 10.1016/j.ijpharm.2009.10.010. Epub 2009 Oct 13.
The success of gene transfer in preclinical animal models and proof of principle clinical studies has made gene therapy an attractive concept for disease treatment. A variety of diseases affecting the lung are candidates for gene therapy. Delivery of genes to the lungs seems to be straightforward, because of the easy accessibility of epithelial cells via the airways. However, efficient delivery and expression of the therapeutic transgene at levels sufficient to result in phenotypic correction of the diseased state have proven elusive. This review presents a brief summary about current status and future prospects in the development of viral and non-viral strategies for pulmonary gene therapy.
基因转移在临床前动物模型中的成功以及原理性临床研究证明，基因治疗是一种有吸引力的疾病治疗概念。许多影响肺部的疾病都适合基因治疗。由于上皮细胞可通过气道轻易接触，因此将基因递送至肺部似乎很简单。然而，高效地递送至肺部并表达治疗性转基因，使其达到足以纠正疾病表型的水平，这一目标一直难以实现。本文简要总结了用于肺部基因治疗的病毒和非病毒策略的最新进展和未来展望。
