Curiel D T, Pilewski J M, Albelda S M
Gene Therapy Program, UAB Comprehensive Cancer Center, University of Alabama at Birmingham, USA.
Am J Respir Cell Mol Biol. 1996 Jan;14(1):1-18. doi: 10.1165/ajrcmb.14.1.8534480.
Gene therapy is the treatment of any disorder or pathophysiologic state based upon the transfer of genetic information. The lung represents a major target of gene therapy for the treatment of genetic disorders such as cystic fibrosis and alpha 1-antitrypsin deficiency. Other diseases are also being targeted, including pulmonary inflammation, surfactant deficiency, pulmonary hypertension, lung cancer, and malignant mesothelioma. This review will examine some general concepts regarding gene transfer and gene therapy, provide an overview of the current vectors being developed to achieve safe and efficient gene transfer, and summarize the ongoing work to apply this new technology to the treatment of both inherited and acquired pulmonary diseases. Although tremendous progress has been made in the ability to successfully transfer genes to cells, there are several unresolved problems limiting the clinical application of this technology to human pulmonary disease. However, as vector technology evolves, gene therapy may become a reality for a number of lung diseases.
基因治疗是基于遗传信息转移对任何疾病或病理生理状态进行的治疗。肺是基因治疗的主要靶点，用于治疗诸如囊性纤维化和α1-抗胰蛋白酶缺乏症等遗传性疾病。其他疾病也在成为治疗目标，包括肺部炎症、表面活性剂缺乏、肺动脉高压、肺癌和恶性间皮瘤。本综述将探讨一些关于基因转移和基因治疗的一般概念，概述目前为实现安全有效的基因转移而研发的载体，并总结将这项新技术应用于遗传性和获得性肺部疾病治疗的正在进行的工作。尽管在成功将基因转移到细胞的能力方面已经取得了巨大进展，但仍有几个未解决的问题限制了这项技术在人类肺部疾病中的临床应用。然而，随着载体技术的发展，基因治疗可能会成为多种肺部疾病的现实治疗手段。
