First-in-human case report: AAV9-hGAA gene therapy for a patient with infantile-onset Pompe disease.
Xiuwei Ma, Jun Li, Xiaodong Wang, Wenhao Ma, Jianhua Wang, Ruijie Gu, Zhiming Zhu, Yongxia Wang, Ying Du, Juan Xu, Fang He, Xiao Yang, Sheng Zhang, Lina Zhu, Qiuping Li, , Zhichun Feng
A single intravenous dose of AAV9-hGAA gene therapy improved motor milestones and GAA enzyme activity in an infant with infantile-onset Pompe disease.
medRxiv · Dec 22, 2022 · PreprintGenetic & Genomic Medicine