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APOE stratified genome-wide association studies provide novel insights into the genetic etiology of Alzheimers's disease

Thomassen, J. Q.; Hampton, L.; Ulms, B.; Grenier-Boley, B.; Heikkinen, S.; Garcia, P.; Castillo-Morales, A.; Kikuchi, M.; Gim, J.; Cao, H.; Kucukali, F.; Amin, N.; Yoon, D.; de Rojas, I.; Alvarez Jerez, P.; Alvarez, V.; Arosio, B.; Bellenguez, C.; Bergh, S.; Billingsley, K.; Blauwendraat, C.; Boada, M.; Borroni, B.; Bossu, P.; Bullido, M. J.; Carracedo, A.; Daniele, A.; de Mendonca, A.; Cookson, M.; Deckert, J.; Dichgans, M.; Djurovic, S.; Dols-Icardo, O.; Dufouil, C.; Duzel, E.; Escott-Price, V.; Fladby, T.; Fratiglioni, L.; Fu, A. K. Y.; Galimberti, D.; Garcia-Alberca, J. M.; Garcia-Ribas, G

2025-05-09 genetic and genomic medicine
10.1101/2025.05.07.25327065 medRxiv
Show abstract

Among the more than 90 identified genetic risk loci for late-onset Alzheimers disease (AD) and related dementias, the apolipoprotein E gene (APOE) {varepsilon}2/{varepsilon}3/{varepsilon}4 polymorphisms remains the longstanding benchmark for genetic disease risk with a consistently large effect across studies1-10. Despite this massive signal, the exact mechanisms for how {varepsilon}4 increases and for how {varepsilon}2 decreases dementia risk is not well-understood. Importantly, recent trials of anti-amyloid therapies suggest less efficacy and higher risks of severe side effects in {varepsilon}4 carriers11-13, hampering the treatment of those with the highest unmet need. To improve our understanding of the genetic architecture of AD in the context of its main genetic driver, we performed genome-wide association studies (GWASs) stratified by {varepsilon}4 and {varepsilon}2 carrier status. Such insights may help to understand and overcome side effects, to impact clinical trial enrolment strategies, and to create the scientific basis for targeted mechanism-driven therapies in neurodegenerative diseases.

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