Xi Qin, Qikun Yu, Xiang Li, Wei Jiang, Xinchang Shi, Wenxiu Hou, Da Zhang, Zhenzhen Cai, Hua Bi, Wenhong Fan, Youxue Ding, Yichen Yang, Biao Dong, Long Chen, DE-Hua Huo, Cong Wang, Yong Zhou, Dening Pei, Miao Ye, Chenggang Liang
Currently, adeno-associated virus (AAV) is one of the primary gene delivery vectors in gene therapy, facilitating long-term in vivo gene expression. Despite being imperative, it is incredibly challenging to precisely assess AAV particle distribution according to the sedimentation coefficient and identify impurities related to capsid structures. This study performed the systematic methodological validation of quantifying the AAV empty and full capsid ratio. This includes specificity, accuracy, precision, linearity, and parameter variables involving the sedimentation velocity analytical ultracentrifugation (SV-AUC) method...
May 8, 2024: Human Gene Therapy