Wu Jun, Li Ren-Ke
Toronto General Research Institute, Division of Cardiovascular Surgery, University Health Network, Toronto, Ontario, Canada.
Division of Cardiac Surgery, Department of Surgery, University of Toronto, Toronto, Ontario, Canada.
Genes Dis. 2016 Aug 20;4(2):64-74. doi: 10.1016/j.gendis.2016.08.001. eCollection 2017 Jun.
Human gene therapy has made significant advances in less than two decades. Within this short period of time, gene therapy has proceeded from the conceptual stage to technology development and laboratory research, and finally to clinical trials for the treatment of a variety of deadly diseases. Cardiovascular disease, cancer, and stroke are leading causes of death worldwide. Despite advances in medical, interventional, radiation and surgical treatments, the mortality rate remains high, and the need for novel therapies is great. Gene therapy provides an efficient approach to disease treatment. Notable advances in gene therapy have been made for genetic disorders, including severe combined immune deficiency, chronic granulomatus disorder, hemophilia and blindness, as well as for acquired diseases, including cancer and neurodegenerative and cardiovascular diseases. However, lack of an efficient delivery system to target cells as well as the difficulty of sustained expression of transgenes has hindered advancements in gene therapy. Ultrasound targeted microbubble destruction (UTMD) is a promising approach for target-specific gene delivery, and it has been successfully investigated for the treatment of many diseases in the past decade. In this paper, we review UTMD-mediated gene delivery for the treatment of cardiovascular diseases, cancer and stroke.
在不到二十年的时间里，人类基因治疗取得了重大进展。在如此短的时间内，基因治疗已从概念阶段发展到技术开发和实验室研究，最终进入治疗各种致命疾病的临床试验阶段。心血管疾病、癌症和中风是全球主要的死亡原因。尽管在医学、介入、放射和外科治疗方面取得了进展，但死亡率仍然很高，对新型疗法的需求也很大。基因治疗为疾病治疗提供了一种有效的方法。基因治疗在遗传性疾病（包括严重联合免疫缺陷、慢性肉芽肿病、血友病和失明）以及后天性疾病（包括癌症、神经退行性疾病和心血管疾病）方面取得了显著进展。然而，缺乏有效的靶向细胞递送系统以及转基因持续表达的困难阻碍了基因治疗的进展。超声靶向微泡破坏（UTMD）是一种有前景的靶向特异性基因递送方法，在过去十年中已成功用于多种疾病的治疗研究。在本文中，我们综述了UTMD介导的基因递送在治疗心血管疾病、癌症和中风方面的应用。
