1 Department of Gynecology and Obstetrics, Development and Related Diseases of Women and Children, Key Laboratory of Sichuan Province, Key Laboratory of Birth Defects and Related Diseases of Women and Children, Ministry of Education, West China Second Hospital, and State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, China .
2 Laboratory of Aging Research and Nanotoxicology, State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, Sichuan, China .
Hum Gene Ther. 2018 Nov;29(11):1227-1234. doi: 10.1089/hum.2018.069. Epub 2018 Sep 5.
Considerable efforts have been devoted to develop safe and efficient gene therapies for life-threatening or inherited diseases. The choice of gene delivery vehicle plays key roles in enhancing the therapeutic effect of nucleic acid cargo. To date, gene therapy approaches involving both viral vectors and nonviral vectors have been evaluated in clinical trials. With improvements in material science and nanotechnologies, positively charged nanoparticles have emerged as potential gene delivery vehicles. In this review, we highlight clinical trials that examined cationic nanocarrier-mediated gene therapy as well as discuss both the toxicity and efficacy of nanocarrier-based therapeutics.
人们投入了大量精力来开发安全有效的基因疗法，以治疗危及生命或遗传性疾病。基因传递载体的选择在提高核酸载药的治疗效果方面起着关键作用。迄今为止，涉及病毒载体和非病毒载体的基因治疗方法已经在临床试验中进行了评估。随着材料科学和纳米技术的进步，带正电荷的纳米颗粒已成为潜在的基因传递载体。在这篇综述中，我们重点介绍了评估阳离子纳米载体介导的基因治疗的临床试验，并讨论了基于纳米载体的治疗的毒性和疗效。
