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.
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| Title: | 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. |
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| Authors: | Epstein, Benjamin E.1 (AUTHOR), Soden, Gabrielle M.1 (AUTHOR), Incao, Arturo A.1 (AUTHOR), Mylvara, Avani1 (AUTHOR), Flynn, Jonathan2 (AUTHOR), Ozsolak, Fatih2 (AUTHOR), Pavan, William J.1 (AUTHOR) wjpavan@gmail.com |
| Source: | PLoS ONE. 3/11/2026, Vol. 21 Issue 3, p1-20. 20p. |
| Database: | Academic Search Ultimate |
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| ISSN: | 19326203 |
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| DOI: | 10.1371/journal.pone.0331275 |
