Cardiovascular Research Institute, Icahn School of Medicine at Mount Sinai, 1470 Madison Avenue, New York, NY 10029, USA.
Department of Pulmonary Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, Vrije Universiteit Amsterdam, 1081 HV Amsterdam, The Netherlands.
Cells. 2022 Mar 12;11(6):984. doi: 10.3390/cells11060984.
Over the past decades, a better understanding of the genetic and molecular alterations underlying several respiratory diseases has encouraged the development of new therapeutic strategies. Gene therapy offers new therapeutic alternatives for inherited and acquired diseases by delivering exogenous genetic materials into cells or tissues to restore physiological protein expression and/or activity. In this review, we review (1) different types of viral and non-viral vectors as well as gene-editing techniques; and (2) the application of gene therapy for the treatment of respiratory diseases and disorders, including pulmonary arterial hypertension, idiopathic pulmonary fibrosis, cystic fibrosis, asthma, alpha-1 antitrypsin deficiency, chronic obstructive pulmonary disease, non-small-cell lung cancer, and COVID-19. Further, we also provide specific examples of lung-targeted therapies and discuss the major limitations of gene therapy.
在过去的几十年中，对几种呼吸系统疾病的遗传和分子改变的更好理解，鼓励了新的治疗策略的发展。通过将外源遗传物质递送到细胞或组织中，基因治疗为遗传性和获得性疾病提供了新的治疗选择，以恢复生理蛋白的表达和/或活性。在这篇综述中，我们回顾了（1）不同类型的病毒和非病毒载体以及基因编辑技术；以及（2）基因治疗在治疗呼吸系统疾病和障碍中的应用，包括肺动脉高压、特发性肺纤维化、囊性纤维化、哮喘、α-1 抗胰蛋白酶缺乏症、慢性阻塞性肺疾病、非小细胞肺癌和 COVID-19。此外，我们还提供了肺靶向治疗的具体例子，并讨论了基因治疗的主要限制。
