The success of recent clinical trials of disease-modifying therapies targeting Aβ have been hampered by lack of brain amyloid pathology in clinically diagnosed AD participants (Salloway et al., 2014) and in future it is likely that many trials of potential disease modifying agents will utilize biomarkers such as CSF amyloid and tau and amyloid PET measures of pathology in participant selection. The gene discussed is MAPT; the disease is Alzheimer disease.