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

Maguire AS, Ta L, Gross AL, Osterhoudt DE, Cannon JS, Hall PI, Sandey M, Seyfried TN, Gray-Edwards HL, Sena-Esteves M, Martin DR. Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease. bioRxiv. 2024 Nov 18.

View in: PubMed