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.
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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An: 192223318
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  Data: 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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  Data: <searchLink fieldCode="AR" term="%22Epstein%2C+Benjamin+E%2E%22">Epstein, Benjamin E.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Soden%2C+Gabrielle+M%2E%22">Soden, Gabrielle M.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Incao%2C+Arturo+A%2E%22">Incao, Arturo A.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Mylvara%2C+Avani%22">Mylvara, Avani</searchLink><relatesTo>1</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Flynn%2C+Jonathan%22">Flynn, Jonathan</searchLink><relatesTo>2</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Ozsolak%2C+Fatih%22">Ozsolak, Fatih</searchLink><relatesTo>2</relatesTo> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Pavan%2C+William+J%2E%22">Pavan, William J.</searchLink><relatesTo>1</relatesTo> (AUTHOR)<i> wjpavan@gmail.com</i>
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  Data: <searchLink fieldCode="JN" term="%22PLoS+ONE%22">PLoS ONE</searchLink>. 3/11/2026, Vol. 21 Issue 3, p1-20. 20p.
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=asn&AN=192223318
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        Value: 10.1371/journal.pone.0331275
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              Text: 3/11/2026
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