Hidai Chiaki, Kitano Hisataka
Division of Physiology, Nihon University School of Medicine, 30-1 Oyaguchikami-cho, Itabashi-ku, Tokyo 173-8610, Japan.
Division of Oral Surgery, Nihon University School of Medicine, 30-1 Oyaguchikami-cho, Itabashi-ku, Tokyo 173-8610, Japan.
Diseases. 2018 Jul 3;6(3):57. doi: 10.3390/diseases6030057.
Although the development of effective viral vectors put gene therapy on the road to commercialization, nonviral vectors show promise for practical use because of their relative safety and lower cost. A significant barrier to the use of nonviral vectors, however, is that they have not yet proven effective. This apparent lack of interest can be attributed to the problem of the low gene transfer efficiency associated with nonviral vectors. The efficiency of gene transfer via nonviral vectors has been reported to be 1/10th to 1/1000th that of viral vectors. Despite the fact that new gene transfer methods and nonviral vectors have been developed, no significant improvements in gene transfer efficiency have been achieved. Nevertheless, some notable progress has been made. In this review, we discuss studies that report good results using nonviral vectors in vivo in animal models, with a particular focus on studies aimed at in vivo gene therapy to treat cancer, as this disease has attracted the interest of researchers developing nonviral vectors. We describe the conditions in which nonviral vectors work more efficiently for gene therapy and discuss how the goals might differ for nonviral versus viral vector development and use.
尽管有效的病毒载体的发展使基因治疗走上了商业化道路,但非病毒载体因其相对安全性和较低成本而显示出实际应用的前景。然而,使用非病毒载体的一个重大障碍是它们尚未被证明有效。这种明显缺乏兴趣可归因于与非病毒载体相关的基因转移效率低的问题。据报道,通过非病毒载体进行基因转移的效率是病毒载体的十分之一到千分之一。尽管已经开发了新的基因转移方法和非病毒载体,但基因转移效率并未取得显著提高。然而,已经取得了一些显著进展。在这篇综述中,我们讨论了在动物模型体内使用非病毒载体报告良好结果的研究,特别关注旨在进行体内基因治疗以治疗癌症的研究,因为这种疾病吸引了开发非病毒载体的研究人员的兴趣。我们描述了非病毒载体在基因治疗中更有效地发挥作用的条件,并讨论了非病毒载体与病毒载体开发和使用的目标可能有何不同。