AAVone: A cost-effective, single-plasmid solution for efficient AAV production with reduced DNA impurities

作者：Rongze Yang, Ngoc Tam Tran, Taylor Chen, Mengtian Cui, Yuyan Wang, Tapan Sharma, Yun Liu, Jiantao Zhang, Xinxu Yuan, Dengwei Zhang, Cuiping Chen, Zhen Shi, Lemin Wang, Yuling Dai, Haniya Zaidi, Jiarui Liang, May Chen, Dabbu Kumar Jaijyan, Huan Hu, Bing Wang, Cheng Xu, Wenhui Hu, Guangping Gao, Daozhan Yu, Phillip W.L. Tai, Qizhao Wang · 发表于：Molecular Therapy — Nucleic Acids · 年份：2025 · DOI：10.1016/j.omtn.2025.102563 · 被引用次数：9 · 研究领域：Virus-based gene therapy research、Viral Infectious Diseases and Gene Expression in Insects、Cancer Research and Treatments

transduction efficiency, but a substantial reduction in DNA impurities from plasmid bacterial backbones and a marked reduction in non-functional snap-back genomes. The AAVone system does not pose a risk for generating replication-competent AAV contaminants. Furthermore, the AAVone system requires significantly less DNAs for AAV production, while achieving favorable full:empty particle ratios and further reducing impurities. In summary, the AAVone platform is a highly efficient, straightforward, cost-effective, and highly consistent AAV production system, making it particularly suitable for manufacturing of GMP-grade AAV vectors.