腺相关病毒载体及其在基因治疗研究中的应用  

Adeno-Associated Virus Vectors and Their Application in Gene Therapy Research

作　　者：陈永昌 刘小平[1,2] 肖浦豪[1,2,4] CHEN Yongchang;LIU Xiaoping;XIAO Puhao(Institute of Primate Translational Medicine,Kunming University of Science and Technology,Kunming 650500,China;State Key Laboratory of Primate Biomedical Research,Kunming University of Science and Technology,Kunming 650500,China;Faculty of Life Science and Technology,Kunming University of Science and Technology,Kunming 650500,China;Southwest United Graduate School,Kunming 650092,China)

机构地区：[1]昆明理工大学灵长类转化医学研究院,云南昆明650500 [2]昆明理工大学省部共建非人灵长类生物医学国家重点实验室,云南昆明650500 [3]昆明理工大学生命科学与技术学院,云南昆明650500 [4]西南联合研究生院,云南昆明650092

出　　处：《昆明理工大学学报（自然科学版）》2024年第3期188-201,共14页Journal of Kunming University of Science and Technology(Natural Science)

基　　金：国家自然科学基金重点项目(81930121).

摘　　要：基因治疗作为生物医药最尖端的领域之一,为人类遗传疾病的精准医疗提供了新途径.通过载体将基因治疗药物高效递送至靶组织或者靶器官是基因治疗成功的关键.腺相关病毒(Adeno-Associated Virus,AAV)因具备较高的安全性、稳定性和组织靶向性以及较低的免疫原性,使其在基因治疗中得以广泛应用.当前,基于AAV载体开发的基因治疗药物已有数款上市,针对不同疾病、靶向特定组织器官的AAV载体也在不断开发中,为未来各类疾病的基因治疗提供了新的可选载体.对AAV的发现与发展历程、生物学特性、载体开发及制备、关键性成药指标等方面开展综述,总结了AAV载体在临床疾病治疗中的应用,展望了AAV载体可能的改造方向,以期为AAV载体开发和应用提供参考.Gene therapy,as one of the most cutting-edge fields in biomedicine,has provided a new approach for precision medicine in the treatment of human genetic diseases.Efficient delivery of gene therapy drugs to target tissues or organs through vectors is key to the success of gene therapy.Adeno-associated virus(AAV)has been widely applied in gene therapy due to its high safety,stability,tissue targeting and low immunogenicity.Currently,several gene therapy drugs based on AAV vectors have been marketed,and AAV vectors targeting specific tissues and organs for different diseases are continuously being developed,providing new optional vectors for gene therapy of various diseases in the future.This paper reviews the discovery and development history,biological characteristics,vector development and preparation,and key druggability indicators of AAV,summarizes the application of AAV vectors in clinical disease treatment,and looks forward to the possible modification directions of AAV vectors,in order to provide references for the development and application of AAV vectors.

关 键 词：基因治疗 腺相关病毒 病毒载体 衣壳工程 

分 类 号：Q782[生物学—分子生物学]