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.
Maguire AS, et al. Among authors: cannon js.
bioRxiv [Preprint]. 2024 Nov 18:2024.11.15.623838. doi: 10.1101/2024.11.15.623838.
bioRxiv. 2024.
PMID: 39605340
Free PMC article.
Preprint.