Systemic neonatal AAV9 gene therapy delivery improves behavioral and phenotypic outcomes comparable to intracerebroventricular delivery in a mouse model of Niemann-Pick disease, type C1
Benjamin E. Epstein, Gabrielle M. Soden, Arturo A. Incao, Avani V. Mylvara, Jonathan R. Flynn, Fatih Ozsolak, William J. Pavan
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.
bioRxiv · Aug 15, 2025 · PreprintNeuroscience