Life-limiting peripheral organ dysfunction in feline Sandhoff disease emerges after effective CNS gene therapy.
Aime K JohnsonVictoria J McCurdyHeather L Gray-EdwardsAnne S MaguireJ Nicholas CochranAmanda L GrossHaleigh E SkinnerAshley N RandleJamie L ShirleyBrandon L BrunsonAllison M BradburyStanley G LeroyMisako HwangHannah E RockwellNancy R CoxHenry J BakerThomas N SeyfriedMiguel Sena-EstevesDouglas R MartinPublished in: Annals of neurology (2023)
The vector formulation in the current study effectively treats neuropathology in feline Sandhoff disease, but whole-body targeting will be an important consideration in next-generation approaches. This article is protected by copyright. All rights reserved.