摘要
基因治疗在临床转化和应用方面取得了许多瞩目的进展。然而,基因治疗面临的主要挑战仍是如何安全有效地递送核酸药物,尤其是实现对靶细胞的按需递送。基于疾病部位异常的氧化还原环境,研究者设计了众多氧化还原响应型核酸药物递送系统。该类递送系统中的氧化还原敏感基团在被氧化或被还原后触发结构断裂或性质转变,从而实现核酸药物在疾病部位的特异性释放,提高疗效。综述了各类氧化还原响应型核酸递送系统的设计思路和原理,并探讨其在肿瘤、炎症等治疗中的应用和发展前景,以期为核酸药物递送系统的发展和临床转化提供参考。
Gene therapy has made great progress in clinical transformation and application. However, the main challenge for gene therapy remains how to deliver nucleic acid drugs safely and effectively, especially to achieve on-demand delivery to target cells. Researchers have designed numerous redox-responsive nucleic acid delivery systems based on the abnormal redox environment at the lesion site. The redoxsensitive groups in the delivery systems undergo structural breaks or property changes after being oxidized or reduced, thereby enabling nucleic acid drugs to be released specifically at the lesion site and improving the efficacy. In this article, we reviewed the design ideas and principles of various redox-responsive nucleic acid delivery systems, as well as discussed their application and development prospects in the treatment of tumors and inflammation, aiming to provide references for the development and clinical transformation of nucleic acid drug delivery systems.
作者
王薪默
曾朝旺
陈旺
刘潇璇
朱丹丹
WANG Xinmo;ZENG Chaowang;CHEN Wang;LIU Xiaoxuan;ZHU Dandan(State Key Laboratory of Natural Medicines,China Pharmaceutical University,Nanjing 210009,China;Center of Advanced Pharmaceuticals and Biomaterials,Institute of Pharmaceutical Sciences,China Pharmaceutical University,Nanjing 210009,China)
出处
《药学进展》
CAS
2022年第11期826-838,共13页
Progress in Pharmaceutical Sciences
基金
国家重点研发计划“政府间国际科技创新合作/港澳台科技创新合作”重点专项项目(No.2018YFE0117800)
国家自然科学基金(No.51773227,No.81701815)
江苏省自然科学基金(No.BK20170735)
中国药科大学天然药物活性组分与药效国家重点实验室资助项目(No.SKLNMZZ202007)。