摘要
腺相关病毒(AAV)作为基因治疗领域中的重要载体,其衣壳蛋白的血清型多样性对于病毒的组织特异性、免疫逃逸能力和基因传递效率具有显著影响。血清型的准确鉴定对于提高基因治疗的安全性和有效性至关重要。本文旨在综合分析当前用于AAV载体衣壳蛋白血清型鉴定的方法,并探讨其在基因疗法中的应用前景。通过系统性文献回顾,重点总结基于质谱法、酶联免疫吸附法、毛细管电泳法以及差示扫描荧光法等不同领域的AAV衣壳蛋白血清型鉴定技术。随着新技术的发展和现有技术的改进,未来的研究将能够更快速、更准确地鉴定AAV血清型,从而推动基因治疗领域的发展。
Adeno-associated virus(AAV),important vector in gene therapy,exhibits serotype diversity in its capsid proteins that significantly influence its tissue tropism,immune evasion capabilities,and gene transfer efficiency.Accurate serotype identification is important for ensuring the safety and efficacy of gene therapy products.This review aims to provide a comprehensive analysis of current methods used for the serotype identification of AAV capsid proteins,and to explore their application prospects in gene therapy.A systematic literature review has been conducted focusing on summarizing AAV capsid protein serotype identification techniques from various fields,including mass spectrometry,enzyme-linked immunosorbent assay,capillary electrophoresis,and differential scanning fluorimetry.With the future development of new technologies and improvements in existing ones,it is possible to identify AAV serotypes more quickly and accurately,thereby promoting the R&D of AAV based gene therapy products.
作者
韩春乐
王可心
王书瑞
杨英
饶春明
HAN Chun-le;WANG Ke-xin;WANG Shu-rui;YANG Ying;RAO Chun-ming(JOINN Drug Quality Research and Testing(Beijing)Co.,Ltd.,Beijing 102600,China;Harbin Medical University Cancer Hospital,Harbin 150081,China)
出处
《药物分析杂志》
北大核心
2025年第4期543-553,共11页
Chinese Journal of Pharmaceutical Analysis
基金
2023年度国家重点研发计划“前沿生物技术专项”——病毒载体的质量研究和质量体系建设(2023YFC3403305)。
关键词
腺相关病毒
基因治疗
衣壳蛋白
血清型鉴定
质谱
毛细管电泳
酶联免疫吸附法
差示扫描荧光法
adeno-associated virus
gene therapy
capsid protein
serotype identification
mass spectrometry
capillary electrophoresis
enzyme-linked immunosorbent assay
differential scanning fluorimetry