- Ohnishi, Takayuki;
- Yanazawa, Masako;
- Sasahara, Tomoya;
- Kitamura, Yasuki;
- Hiroaki, Hidekazu;
- Fukazawa, Yugo;
- Kii, Isao;
- Nishiyama, Takashi;
- Kakita, Akiyoshi;
- Takeda, Hiroyuki;
- Takeuchi, Akihide;
- Arai, Yoshie;
- Ito, Akane;
- Komura, Hitomi;
- Hirao, Hajime;
- Satomura, Kaori;
- Inoue, Masafumi;
- Muramatsu, Shin-ichi;
- Matsui, Ko;
- Tada, Mari;
- Sato, Michio;
- Saijo, Eri;
- Shigemitsu, Yoshiki;
- Sakai, Satoko;
- Umetsu, Yoshitaka;
- Goda, Natsuko;
- Takino, Naomi;
- Takahashi, Hitoshi;
- Hagiwara, Masatoshi;
- Sawasaki, Tatsuya;
- Iwasaki, Genji;
- Nakamura, Yu;
- Nabeshima, Yo-ichi;
- Teplow, David B;
- Hoshi, Minako
Neurodegeneration correlates with Alzheimer's disease (AD) symptoms, but the molecular identities of pathogenic amyloid β-protein (Aβ) oligomers and their targets, leading to neurodegeneration, remain unclear. Amylospheroids (ASPD) are AD patient-derived 10- to 15-nm spherical Aβ oligomers that cause selective degeneration of mature neurons. Here, we show that the ASPD target is neuron-specific Na(+)/K(+)-ATPase α3 subunit (NAKα3). ASPD-binding to NAKα3 impaired NAKα3-specific activity, activated N-type voltage-gated calcium channels, and caused mitochondrial calcium dyshomeostasis, tau abnormalities, and neurodegeneration. NMR and molecular modeling studies suggested that spherical ASPD contain N-terminal-Aβ-derived "thorns" responsible for target binding, which are distinct from low molecular-weight oligomers and dodecamers. The fourth extracellular loop (Ex4) region of NAKα3 encompassing Asn(879) and Trp(880) is essential for ASPD-NAKα3 interaction, because tetrapeptides mimicking this Ex4 region bound to the ASPD surface and blocked ASPD neurotoxicity. Our findings open up new possibilities for knowledge-based design of peptidomimetics that inhibit neurodegeneration in AD by blocking aberrant ASPD-NAKα3 interaction.