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.