Astrocyte-targeted gene therapy demonstrates safety and efficacy in two murine models of Vanishing White Matter disease

J. Bonkowsky, J. Herstine, P-K Chang, S. Chornyy, K. Nokhrina, J. Redinger, J. Wentz,N. Pyne, C. Holaway, A. Sunshine, E. Scholl,T. Vetter,T. Stevenson,K. Flanigan,A. Bradbury

ANNALS OF NEUROLOGY(2023)

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Neurogenetics, Experimental Therapeutics
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