Park Tae Gwan, Jeong Ji Hoon, Kim Sung Wan
Department of Biological Sciences, Korea Advanced Institute of Science and Technology, Daejeon 305-701, Korea.
Adv Drug Deliv Rev. 2006 Jul 7;58(4):467-86. doi: 10.1016/j.addr.2006.03.007. Epub 2006 Jun 15.
Gene therapy provides great opportunities for treating diseases from genetic disorders, infections and cancer. To achieve successful gene therapy, development of proper gene delivery systems could be one of the most important factors. Several non-viral gene transfer methods have been developed to overcome the safety problems of their viral counterpart. Polymer-based non-viral gene carriers have been used due to their merits in safety including the avoidance of potential immunogenecity and toxicity, the possibility of repeated administration, and the ease of the establishment of good manufacturing practice (GMP). A wide range of polymeric vectors have been utilized to deliver therapeutic genes in vivo. The modification of polymeric vectors has also shown successful improvements in achieving target-specific delivery and in promoting intracellular gene transfer efficiency. Various systemic and cellular barriers, including serum proteins in blood stream, cell membrane, endosomal compartment and nuclear membrane, were successfully circumvented by designing polymer carriers having a smart molecular structure. This review explores the recent development of polymeric gene carriers and presents the future directions for the application of the polymer-based gene delivery systems in gene therapy.
基因治疗为治疗遗传性疾病、感染性疾病和癌症等提供了巨大机遇。为实现成功的基因治疗，开发合适的基因递送系统可能是最重要的因素之一。为克服病毒载体的安全问题，人们已开发出多种非病毒基因转移方法。基于聚合物的非病毒基因载体因其在安全性方面的优点而被使用，这些优点包括避免潜在的免疫原性和毒性、可重复给药以及易于建立良好生产规范（GMP）。多种聚合物载体已被用于在体内递送治疗性基因。聚合物载体的修饰在实现靶向特异性递送和提高细胞内基因转移效率方面也显示出成功的改进。通过设计具有智能分子结构的聚合物载体，成功规避了各种系统性和细胞性屏障，包括血流中的血清蛋白、细胞膜、内体区室和核膜。本综述探讨了聚合物基因载体的最新进展，并提出了基于聚合物的基因递送系统在基因治疗中的应用未来方向。
