Using an In Vivo Mouse Model to Determine the Exclusion Criteria of Preexisting Anti-AAV9 Neutralizing Antibody Titer of Pompe Disease Patients in Clinical Trials

Using an In Vivo Mouse Model to Determine the Exclusion Criteria of Preexisting Anti-AAV9 Neutralizing Antibody Titer of Pompe Disease Patients in Clinical Trials

The efficacy of adeno-associated virus (AAV)-based gene therapy is dependent on effective viral transduction, which might be inhibited by preexisting immunity to AAV acquired from infection or maternal delivery. Anti-AAV neutralizing Abs (NAbs) titer is usually measured by in vitro assay and used fo...

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Bibliographic Details
Main Authors: Hanqing Wang, Cengceng Zhang, Zheyue Dong, Xueyang Zhu, Xuchu Zheng, Ziyang Liu, Jianfang Zhou, Shuangqing Yu, Xiaobing Wu, Xiaoyan Dong
Format: Article
Language:English
Published: MDPI AG 2024-03-01
Series:Viruses
Subjects:
gene therapy
adeno-associated virus
preexisting neutralizing antibody
Pompe disease
Online Access:https://www.mdpi.com/1999-4915/16/3/400

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