AHA1 Regulates Aβ Production via Modulation of APP Abundance and γ-Secretase Assembly

Summary

Deposition of amyloid β-protein (Aβ) is a hallmark of Alzheimer's disease (AD), produced by γ-secretase-mediated cleavage of amyloid precursor protein (APP). The 90-kDa heat shock protein (Hsp90) co-chaperone, activator of Hsp90 ATPase homolog 1 (AHA1), is known to promote the accumulation of toxic tau species; however, its effects on Aβ production remain unclear. Using cellular models-including human embryonic kidney 293 T (HEK293T) cells, stable HEK-APP cells, presenilin 1/2 double-knockout mouse embryonic fibroblasts (PS-DKO MEFs), and Chinese hamster ovary (CHO) cells-in which AHA1 was experimentally manipulated, together with observational analyses of human iPSC-derived neurons carrying an FAD-linked APP mutation, we demonstrate that AHA1 regulates Aβ generation through two mechanisms: regulation of APP protein abundance through pathways that are at least partially independent of Hsp90 interaction and Hsp90-dependent promotion of γ-secretase assembly. Knockdown of endogenous AHA1 reduces Aβ production and decreases APP and γ-secretase component levels, whereas AHA1 overexpression elevates Aβ generation and increases the expression of these proteins. The AHA1-E67K mutant, which has impaired Hsp90 binding, lowers Aβ production and the levels of APP and γ-secretase components compared with wild-type AHA1. AHA1 associates with APP and APH1, an immature γ-secretase component, indicating its role in APP proteolysis and Aβ production. Disruption of the AHA1/Hsp90 complex-through AHA1 knockdown, the E67K mutant, or a small-molecule inhibitor-reduces γ-secretase assembly. Notably, Familial AD mutations (APP-C99-I45F, PS1-L286V) upregulate AHA1/Hsp90, elevating APP/APH1 and Aβ42 production. AHA1 knockdown rescues FAD-linked Aβ42 overproduction in a mutant cell model, while AHA1 overexpression exacerbates PS1 mutant Aβ production. Collectively, these findings reveal that AHA1 regulates Aβ production by modulating APP abundance and γ-secretase assembly, establishing AHA1 as a potential target for therapeutic intervention in AD. © 2026 The Author(s). Journal of Neurochemistry published by John Wiley & Sons Ltd on behalf of International Society for Neurochemistry.

Authors Noorani AA, Islam S, Filip E, Catalfano K, Paswan S, Nagarajan V, Wilkins HM, Blagg BSJ, Zou K, Wolfe MS
Journal Journal of neurochemistry
Publication Date 2026 Sep;170(9):e70544
PubMed 42661397
PubMed Central PMC13522734
DOI 10.1111/jnc.70544

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