AAV-APOE modulation therapy

AAV-APOE modulation therapy is an experimental gene therapy program developed by uniQure for the treatment of Alzheimer's disease and other tauopathies. The therapy utilizes adeno-associated virus (AAV) vectors to deliver genetic payloads to the central nervous system. The approach is dual-pronged: it employs AAV-encoded microRNAs (miRNAs) to silence the expression of the apolipoprotein E4 (APOE4) allele—the primary genetic risk factor for late-onset Alzheimer's—and/or delivers transgenes to express neuroprotective variants such as APOE2. By modulating APOE protein levels, the therapy aims to restore lipid homeostasis and mitigate disease progression. Preclinical studies in mouse models of tauopathy have demonstrated robust modulation of APOE levels, although initial data suggests that APOE modulation alone may not be sufficient to directly reduce pTau181 levels in the absence of concurrent amyloid pathology.

Other names

AAV-mediated APOE modulationAAV-encoded APOE targeting miRNAsAAV-encoded protective APOE transgenes