While acknowledging that there is currently no known animal model which exactly recapitulates human AD, and that often, this preclinical evidence does not translate to the bedside, this review delves into some of the in vitro and in vivo data that exist to support certain therapeutic candidates on the basis of improving AD-associated pathologies, functional disturbances and biomarkers, such as neuronal cell death, neuronal plasticity, Aβ deposition, and tau protein hyperphosphorylation. This evidence concerns the gene MAPT and Alzheimer disease.