Comparison of neonatal systemic and intracerebroventricular AAV9 gene therapy delivery demonstrating improved behavioral and phenotypic outcomes in a mouse model of Niemann-Pick disease, type C1 - Benjamin E. Epstein; Gabrielle M. Soden; Arturo A. Incao; Avani Mylvara; Jonathan Flynn; Fatih Ozsolak; William J. Pavan (PLoS ONE, 2026)