NeurosciencebioRxivPreprint
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…·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.
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