Article

Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease

2024-11-18

Abstract excerpt

Sandhoff Disease (SD), a fatal neurodegenerative disorder, is caused by the absence of ß-hexosaminidase (Hex) and subsequent accumulation of GM2 ganglioside in lysosomes. Previous studies have led to adeno-associated virus (AAV) gene therapy for children with GM2 gangliosidosis in both expanded access and Phase I/II clinical trials via intracranial and/or cerebrospinal fluid-based delivery. The current study inves...

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Literature Corpus work: 479772f5-a10f-53a8-acaf-2f291713b5cb
DOI: 10.1101/2024.11.15.623838

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