Xia Ke, Liu Shuangling, Wu Zhenkun, Jiang Jian-Hui
State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Affiliated Hospital of Hunan university, School of Biomedical Sciences, Hunan University, Changsha, Hunan 410082, China.
Chembiochem. 2025 Apr 1;26(7):e202400856. doi: 10.1002/cbic.202400856. Epub 2025 Jan 9.
Adeno-associated virus (AAV) has emerged as a powerful and effective tool for the delivery of exogenous genes into various cells or tissues. To improve the gene delivery efficiency, as well as the safety and specificity of AAV's cell-targeting capabilities, extensive investigations have been conducted into its molecular biological characteristics, including capsid structure, cellular tropism, and the mechanisms underlying its entry, replication, DNA packaging, and capsid assembly. Significant differences exist between human and non-human primate AAVs regarding tissue targeting and transduction efficiency. These differences are primarily attributed to the amino acid sequences of AAV capsid proteins, the structural characteristics of these proteins, and the interactions of AAV with surface factors on host cells, such as cell surface receptors, signaling molecules, and associated proteins. This review primarily focuses on several key aspects of AAV, including its genome, coat proteins and their structures, genome replication, virus assembly, and the role of helper viruses. Additionally, it examines the utilization of recombinant adeno-associated viruses (rAAV), detailing their production methods, mechanisms of cell entry and trafficking, and various serotypes. The review further interprets the role of rAAV by analyzing its current applications in research and therapy.
腺相关病毒（AAV）已成为一种将外源基因导入各种细胞或组织的强大而有效的工具。为了提高基因传递效率以及AAV细胞靶向能力的安全性和特异性，人们对其分子生物学特性进行了广泛研究，包括衣壳结构、细胞嗜性以及其进入、复制、DNA包装和衣壳组装的潜在机制。人类和非人灵长类动物的AAV在组织靶向和转导效率方面存在显著差异。这些差异主要归因于AAV衣壳蛋白的氨基酸序列、这些蛋白的结构特征以及AAV与宿主细胞表面因子（如细胞表面受体、信号分子和相关蛋白）的相互作用。本综述主要关注AAV的几个关键方面，包括其基因组、衣壳蛋白及其结构、基因组复制、病毒组装以及辅助病毒的作用。此外，还研究了重组腺相关病毒（rAAV）的应用，详细介绍了其生产方法、细胞进入和运输机制以及各种血清型。本综述通过分析rAAV目前在研究和治疗中的应用来进一步阐释其作用。
