AAV-protective APOE

AAV-protective APOE is a gene therapy approach developed by uniQure for the treatment of Alzheimer's disease and other tauopathies. The strategy utilizes adeno-associated virus (AAV) vectors to deliver protective variants of the apolipoprotein E (APOE) gene, such as APOE2, which is known to be neuroprotective and associated with a lower risk of late-onset Alzheimer's. In some experimental configurations, the AAV vector is designed to simultaneously express protective APOE transgenes and deliver microRNA (miRNA) sequences to silence the expression of the risk-associated APOE4 allele. Preclinical data presented at ASGCT 2026 investigated the efficacy of this modulation in transgenic mouse models of tauopathy, finding that while APOE protein levels were successfully modulated, there was no significant reduction in pTau181 levels, suggesting that APOE-related effects on tau pathology may occur upstream or depend on concurrent amyloid pathology.

Other names

AAV-encoded protective APOE variantsAAV-mediated APOE modulation