Xiaobing Wu

This paper describes the use of GC801 to reduce anti-AAV9 antibodies in a neonate with Pompe disease, enabling successful AAV9 gene therapy.

The paper describes a new salamander species, Pachytriton, from Anhui, China, based on morphological and molecular analyses.

A single intravenous dose of AAV9-hGAA gene therapy improved motor milestones and GAA enzyme activity in an infant with infantile-onset Pompe disease.