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@ARTICLE{Maruyama:285051,
author = {Maruyama, Riki and Fukumori, Akio and Funamoto, Satoru and
Okada, Ken and Akamine, Shoshin and Yanagida, Kanta and
Shinohara, Mitsuru and Sato, Naoyuki and Okochi, Masayasu
and Kudo, Takashi and Steiner, Harald},
title = {γ-{S}ecretase exosites as targets for substrate-selective
lowering of {A}β generation.},
journal = {Structure},
volume = {34},
number = {2},
issn = {0969-2126},
address = {London [u.a.]},
publisher = {Elsevier Science},
reportid = {DZNE-2026-00175},
pages = {363 - 374.e4},
year = {2026},
abstract = {Intramembrane proteolysis by γ-secretase is critically
implicated in Alzheimer disease pathogenesis by processing
of its amyloid precursor protein substrate C99 into harmful
amyloid-β peptide (Aβ) species. Recruitment of C99
involves binding of its N-terminal extracellular domain to
exosites in γ-secretase. However, the role of these
interactions has been elusive. Here, we show that the
N-terminally shorter extracellular domain of the
non-amyloidogenic C83 substrate also interacts with
γ-secretase exosites, but more weakly. Moreover, we found
that bulky aromatic mutations within the 16 amino acid
extension of C99 interfere with exosite binding and inhibit
substrate cleavage. Likewise, peptides binding to the C99
N-terminus that selectively inhibit Aβ production in vitro
and in vivo interfere with exosite binding of C99. Our data
show that exosite interactions of the C99 N-terminal region
with γ-secretase can impact substrate cleavage and indicate
that interfering with exosite interactions of C99 may
provide a means for modulating amyloidogenic substrate
processing.},
keywords = {Amyloid Precursor Protein Secretases: metabolism / Amyloid
Precursor Protein Secretases: chemistry / Amyloid Precursor
Protein Secretases: genetics / Humans / Protein Binding /
Amyloid beta-Protein Precursor: metabolism / Amyloid
beta-Protein Precursor: chemistry / Amyloid beta-Protein
Precursor: genetics / Substrate Specificity / Amyloid
beta-Peptides: metabolism / Binding Sites / Proteolysis /
Peptide Fragments: metabolism / Peptide Fragments: chemistry
/ Peptide Fragments: genetics / Models, Molecular / Protein
Domains / Mutation / Animals / HEK293 Cells / Alzheimer
Disease: metabolism / APP (Other) / Alzheimer's disease
(Other) / C83 (Other) / C99 (Other) / amyloid-β peptide
(Other) / exosite (Other) / peptide (Other) / γ-secretase
(Other) / Amyloid Precursor Protein Secretases (NLM
Chemicals) / Amyloid beta-Protein Precursor (NLM Chemicals)
/ Amyloid beta-Peptides (NLM Chemicals) / Peptide Fragments
(NLM Chemicals)},
cin = {AG Steiner},
ddc = {540},
cid = {I:(DE-2719)1110000-1},
pnm = {352 - Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-352},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41418776},
doi = {10.1016/j.str.2025.11.010},
url = {https://pub.dzne.de/record/285051},
}