基因治疗药物递送系统研究进展

Research Progress in Delivery Systems for Gene Therapy Agents

基因治疗在癌症及许多遗传性疾病治疗方面有着重要意义。由于基因药物在体内很容易降解,为了有效地将基因药物递送至靶组织、靶细胞及靶细胞器发挥作用,开发安全高效的基因药物载体体系是十分必要的。随着基因治疗的发展和进一步完善,各类非病毒和病毒载体材料应运而生。针对各类基于非病毒和病毒载体的基因递送系统研究及其临床试验中的进展进行汇总。

Gene therapy holds important implications for the treatment of cancer and numerous genetic diseases. Due to their easy degradation by serum enzymes in vivo, it is necessary to develop safe and effective delivery systems for gene therapy products so that they can be effectively delivered to the target sites including target tissues, target cells and target organelles. In response to the development and maturation of gene therapy, various non-viral and viral carriers have emerged. In this review, we summarized the research and clinical trial progress of various gene delivery systems based on viral and non-viral vectors.