Alzheimer's therapy targeting the β-secretase enzyme BACE1: Benefits and potential limitations from the perspective of animal model studies

M Ohno - Brain research bulletin, 2016 - Elsevier
Accumulating evidence points to the amyloid-β (Aβ) peptide as the culprit in the
pathogenesis of Alzheimer's disease (AD). β-Site amyloid precursor protein (APP)-cleaving
enzyme 1 (BACE1) is a protease that is responsible for initiating Aβ production. Although
precise mechanisms that trigger Aβ accumulation remain unclear, BACE1 inhibition
undoubtedly represents an important intervention that may prevent and/or cure AD.
Remarkably, animal model studies with knockouts, virus-delivered small interfering RNAs …