Gautam Ajay, Waldrep J Clifford, Densmore Charles L
Department of Molecular Physiology, Baylor College of Medicine, 1 Baylor Plaza, Houston, TX 77030, USA.
Mol Biotechnol. 2003 Jan;23(1):51-60. doi: 10.1385/mb:23:1:51.
Gene therapy is a novel field of medicine that holds tremendous therapeutic potential for a variety of human diseases. Targeting of therapeutic gene delivery vectors to the lungs can be beneficial for treatment of various pulmonary diseases such as lung cancer, cystic fibrosis, pulmonary hypertension, alpha-1 antitrypsin deficiency, and asthma. Inhalation therapy using formulations delivered as aerosols targets the lungs through the pulmonary airways. The instant access and the high ratio of the drug deposited within the lungs noninvasively are the major advantages of aerosol delivery over other routes of administration. Delivery of gene formulations via aerosols is a relatively new field, which is less than a decade old. However, in this short period of time significant developments in aerosol delivery systems and vectors have resulted in major advances toward potential applications for various pulmonary diseases. This article will review these advances and the potential future applications of aerosol gene therapy technology.
基因治疗是医学领域的一个新兴领域,对多种人类疾病具有巨大的治疗潜力。将治疗性基因递送载体靶向输送至肺部,可能有益于治疗各种肺部疾病,如肺癌、囊性纤维化、肺动脉高压、α-1抗胰蛋白酶缺乏症和哮喘。使用气雾剂剂型的吸入疗法通过肺气道靶向作用于肺部。气雾剂给药相对于其他给药途径的主要优势在于能够立即起效,且药物能以无创方式高比例沉积于肺内。通过气雾剂递送基因制剂是一个相对较新的领域,其发展历程尚不足十年。然而,在这短短的时间内,气雾剂递送系统和载体取得了显著进展,朝着各种肺部疾病的潜在应用迈出了重要步伐。本文将综述这些进展以及气雾剂基因治疗技术未来的潜在应用。