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Author

Courtney J. Garrett

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Sep 2026

Intravenous gene therapy improves life span and clinical outcomes in a feline model of Sandhoff disease.

Dose-dependent reductions of GM2 ganglioside storage and increases in Hex activity were documented, associated with reduced neuroinflammatory cell populations and partial correction of myelin deficits, supporting potential for translation to patients with SD.

Anne S. Maguire, N. Ta, Amanda Gross et al. · 0 citations

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