Genetic & Genomic MedicinemedRxivPreprint
First-in-human case report: AAV9-hGAA gene therapy for a patient with infantile-onset Pompe disease.
Xiuwei Ma, Jun Li, Xiaodong Wang…·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.
4 citations