腺相关病毒在视觉系统神经示踪及眼科治疗中的应用进展  

Application Progress of Adeno-associated Virus in Nerve Tracing of Visual System and Ophthalmic Treatment

作　　者：蔡丹瑞 沈吟[1,2] CAI Danrui;SHEN Yin(Eye Center,Renmin Hospital of Wuhan University,Wuhan 430060,China;Medical Research Institute,Wuhan University,Wuhan 430071,China)

机构地区：[1]武汉大学人民医院眼科中心,武汉430060 [2]武汉大学医学研究院,武汉430071

出　　处：《医学综述》2020年第22期4440-4444,共5页Medical Recapitulate

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(2017YFE0103400);国家自然科学基金面上项目(81470628)。

摘　　要：腺相关病毒(AAV)属微小病毒科。目前血清型AAV有12种,其因低免疫原性、无致病性、宿主范围广、可长期表达等优点,成为眼科非跨突触病毒示踪和基因治疗中的重要载体,包括携带荧光蛋白后在视觉传导通路中的示踪技术、加入特定元件实现靶向结合基因组修饰、作为病毒载体进行基因治疗等多种应用方式的实现。但AAV载体仍存在一些问题,如对靶向细胞的感染效率和特异性不高以及在临床试验中机体免疫反应较重等。因此,未来仍需对AAV的病毒生物学特性进行深入研究。Adeno-associated virus(AAV)is a member of the parvoviridae,and twelve serotypes have been discovered from humans.AAV as gene transfer vector has become an important carrier in non-transsynaptic virus tracing and gene therapy for eye diseases because of its low immunogenicity,no pathogenicity,broad host range and long-term expression,including the application of fluorescent protein-carrying AAV in visual conduction pathway,the application of adding specific elements to achieve targeted binding genomic modification,used in gene therapy as viral vector and other applications.There are still some problems remained,such as the low efficiency and specificity of infection on targeted cells,high immunoreaction in clinical trials,which require further study of the biological characteristics of AAV.

关 键 词：腺相关病毒 常间回文重复序列丛集/常间回文重复序列丛集关联蛋白系统 基因治疗 

分 类 号：R37[医药卫生—病原生物学]