Juan Xu

GC801-enabled AAV gene therapy in a neonate with Pompe disease

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

Heart spheroids from primary cells for studying cardiac development

This study generates heart spheroids from murine primary cells and shows they retain in vivo cell lineages and respond to growth factors and cell interactions.

Microbes under climatic refugia: Self-stabilizing subcommunity rank dynamics in large-river deltaic estuaries

The paper examines microbial subcommunity dynamics in large-river deltaic estuaries, finding that their rank structures are persistent and resilient to environmental changes.

Cardiac Fibroblasts regulate myocardium and coronary vasculature development via the collagen signaling pathway

This study identifies the role of cardiac fibroblasts in regulating myocardium and coronary vasculature development through the collagen signaling pathway using genetic ablation and single-cell RNA sequencing.

First-in-human case report: AAV9-hGAA gene therapy for a patient with infantile-onset Pompe disease.

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