Wang Qizhao, Lü Yinghui, Li Zhaofa, Diao Yong, Xu Rui'an
Institute of Molecular Medicine, Huaqiao University, Quanzhou 362021, China.
Sheng Wu Gong Cheng Xue Bao. 2011 Apr;27(4):531-8.
Recombinant adeno-associated viral vectors (rAAV) have been widely used as gene therapy vectors in clinical trials. Here, we reviewed the genomic structures and replication mechanisms of wt-AAV. Then, the assembly of capsid and the encapsidation of genomic DNA, two major events during AAV pakaging, was discussed in detail. Although the overall pattern of virus assembly and encapsidation is known, the molecular mechanisms and the structure-function relationship involved in these processes are not well understood. Further elucidatation of these processes may improve the production technology of rAAV and develop gene drug based on rAAV.
重组腺相关病毒载体（rAAV）已在临床试验中广泛用作基因治疗载体。在此，我们综述了野生型AAV的基因组结构和复制机制。然后，详细讨论了衣壳组装和基因组DNA包装这两个AAV包装过程中的主要事件。尽管病毒组装和包装的总体模式是已知的，但这些过程中涉及的分子机制和结构-功能关系尚不清楚。进一步阐明这些过程可能会改进rAAV的生产技术，并开发基于rAAV的基因药物。
