Intravenous gene therapy improves life span and clinical outcomes in a feline model of Sandhoff disease.

Intravenous gene therapy extends lifespan up to nearly three times in a feline model of Sandhoff disease, with dose-dependent improvements in clinical and biochemical outcomes.

• Why it matters: Sandhoff disease is a fatal neurodegenerative disorder lacking effective treatments, and current gene therapy approaches have primarily focused on direct brain delivery, limiting their clinical applicability.
• What they did: Researchers administered a bicistronic AAV vector-based gene therapy intravenously to presymptomatic Felis catus with SD, testing low and high doses and assessing lifespan, neurological function, and biochemical markers.
• The result: The therapy significantly increased lifespan, improved neurological symptoms, reduced GM2 ganglioside storage, and partially normalized brain structure and metabolism, supporting its potential for human translation.