Adeno-Associated Viral (AAV) Vector-Mediated GLB1 Gene Transfer Reduces Substrate Accumulations in a Murine Model of GM1 Gangliosidosis

Yan G. Ni,Michael Gelb,Zachary Herbst, Catherine Vrentas, Elyse Messick, David A. Weinstein, Susan E. Browne, James M. Wilson, Christian Hinderer

MOLECULAR THERAPY(2023)

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