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. - Epstein, Benjamin E.; Soden, Gabrielle M.; Incao, Arturo A.; Mylvara, Avani; Flynn, Jonathan; Ozsolak, Fatih; Pavan, William J. (PLoS ONE, 2026)