Zheng Qianqian, Lin Deqing, Lei Lei, Li Xingyi, Shi Shuai
J Biomed Nanotechnol. 2017 Dec 1;13(12):1565-1580. doi: 10.1166/jbn.2017.2489.
Following the US Food and Drug Administration's approval of gene therapy drugs, the great potential of disease gene therapy is once again being taken seriously. However, finding a safe and efficient gene delivery vector remains the biggest challenge to successful gene therapy. To overcome this, engineered non-viral gene delivery vectors are continually being developed, despite their lower efficiencies compared with viral vectors. However, the complex biological barriers and instability caused by charge interactions mean that, no major breakthroughs have been made in the clinical development of these non-viral gene vectors. Nevertheless, the functions of the engineered polymer gene carriers are continuously expanding, and success has been achieved with the combined use of therapeutic molecules and targeted delivery. These polymer gene-carrier vectors offer a broader choice of new treatments for diseases ever before.
在美国食品药品监督管理局批准基因治疗药物后,疾病基因治疗的巨大潜力再次受到重视。然而,找到一种安全有效的基因递送载体仍然是基因治疗成功的最大挑战。为了克服这一问题,尽管与病毒载体相比效率较低,但工程化非病毒基因递送载体仍在不断开发。然而,复杂的生物屏障以及电荷相互作用导致的不稳定性意味着,这些非病毒基因载体的临床开发尚未取得重大突破。尽管如此,工程化聚合物基因载体的功能在不断扩展,并且在治疗分子与靶向递送的联合应用方面已经取得了成功。这些聚合物基因载体为疾病提供了比以往任何时候都更广泛的新治疗选择。