400 Investigation of a translational astrocyte-targeted AAV-mediated gene addition therapy in two models of Vanishing White Matter disease

400 Investigation of a translational astrocyte-targeted AAV-mediated gene addition therapy in two models of Vanishing White Matter disease

OBJECTIVES/GOALS: Vanishing White Matter Disease (VWM), is a childhood neurodegenerative leukodystrophy that presents with motor deficits, neurologic decline, and seizures leading to death.There are no treatments. Herein we investigate adeno-associated virus serotype 9 (AAV9) gene addition therapy f...

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Bibliographic Details
Main Authors: Jessica A. Herstine, Pi-Kai Chang, Sergiy Chornyy, Tamara J. Stevenson, Jessica Rediger, Julia Wentz, Nettie Pyne, Joshua L. Bonkowsky, Allison M. Bradbury
Format: Article
Language:English
Published: Cambridge University Press 2024-04-01
Series:Journal of Clinical and Translational Science
Online Access:https://www.cambridge.org/core/product/identifier/S2059866124003480/type/journal_article

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https://www.cambridge.org/core/product/identifier/S2059866124003480/type/journal_article

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