Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease
Anne S. Maguire, Nathan L. Ta, Amanda L. Gross, Courtney Garrett, Arthur D. Zimmerman, Elise B. Diffie, Devin E. Osterhoudt, Sara H. Pacer, Morgan L. Mitchell, Jessica S. Cannon, Paige I. Hall, Maninder Sandey, Robert C. Cole, Thomas N. Seyfried, Heather L. Gray‐Edwards, , Douglas R. Martin
Intravenous AAV gene therapy extends lifespan and improves clinical outcomes in feline Sandhoff Disease by reducing GM2 ganglioside accumulation and neuroinflammation.
Science Translational Medicine · Sep 9, 2026Peer-reviewedPathology