Arturo A. Incao

This study evaluates the efficacy of systemic AAV9-hNPC1 gene therapy in NPC1 mouse models, finding that higher doses and earlier treatment improve survival and slow disease progression.

This study compares systemic neonatal and intracerebroventricular AAV9 gene therapy in Npc1-deficient mice, finding that systemic neonatal delivery achieves comparable survival and phenotypic improvements to ICV delivery.