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@ARTICLE{Page:136732,
author = {Page, Richard M and Münch, Anna and Horn, Thomas and Kuhn,
Peer-Hendrik and Colombo, Alessio and Reiner, Orly and
Boutros, Michael and Steiner, Harald and Lichtenthaler,
Stefan F and Haass, Christian},
title = {{L}oss of {PAFAH}1{B}2 reduces amyloid-β generation by
promoting the degradation of amyloid precursor protein
{C}-terminal fragments.},
journal = {The journal of neuroscience},
volume = {32},
number = {50},
issn = {0270-6474},
address = {Washington, DC},
publisher = {Soc.57413},
reportid = {DZNE-2020-03054},
pages = {18204-18214},
year = {2012},
abstract = {Amyloid-β peptide (Aβ) is believed to play a central role
in the pathogenesis of Alzheimer's disease. In view of the
side effects associated with inhibiting the secretases that
produce Aβ, new molecular targets are required to provide
alternative therapeutic options. We used RNA interference
(RNAi) to systematically screen the Drosophila genome to
identify genes that modulate Aβ production upon knockdown.
RNAi of 41 genes in Drosophila cells significantly lowered
Aβ without affecting general secretion or viability. After
the γ-secretase complex components, the most potent effect
was observed for platelet activating factor acetylhydrolase
α (Paf-AHα), and, in mammalian cells, the effect was
replicated for its ortholog PAFAH1B2. Knockdown of PAFAH1B2
strongly reduced Aβ secretion from human cells, and this
effect was confirmed in primary cells derived from PAFAH1B2
knock-out mice. Reduced Aβ production was not attributable
to altered β-amyloid precursor protein (APP) ectodomain
shedding but was a result of an enhanced degradation of APP
C-terminal fragments (CTFs) in the absence of PAFAH1B2 but
not its close homolog PAFAH1B3. Enhanced degradation of APP
CTFs was selective because no such effects were obtained for
Notch or E-/N-cadherin. Thus, we have identified an
important protein that can selectively modify Aβ generation
via a novel mechanism, namely enhanced degradation of its
immediate precursor. In view of the absence of a
neurological phenotype in PAFAH1B2 knock-out mice, targeted
downregulation of PAFAH1B2 may be a promising new strategy
for lowering Aβ.},
keywords = {1-Alkyl-2-acetylglycerophosphocholine Esterase: genetics /
1-Alkyl-2-acetylglycerophosphocholine Esterase: metabolism /
Amyloid beta-Peptides: metabolism / Amyloid beta-Protein
Precursor: metabolism / Animals / Drosophila / Gene
Knockdown Techniques / HEK293 Cells / Humans / Mice / Mice,
Knockout / Microtubule-Associated Proteins: genetics /
Microtubule-Associated Proteins: metabolism / Peptide
Fragments: metabolism / RNA Interference / Transfection /
Amyloid beta-Peptides (NLM Chemicals) / Amyloid beta-Protein
Precursor (NLM Chemicals) / Microtubule-Associated Proteins
(NLM Chemicals) / Peptide Fragments (NLM Chemicals) /
1-Alkyl-2-acetylglycerophosphocholine Esterase (NLM
Chemicals) / PAFAH1B1 protein, human (NLM Chemicals)},
cin = {AG Lichtenthaler / AG Steiner / AG Haass old},
ddc = {610},
cid = {I:(DE-2719)1110006 / I:(DE-2719)1110000-1 /
I:(DE-2719)1110007},
pnm = {342 - Disease Mechanisms and Model Systems (POF3-342)},
pid = {G:(DE-HGF)POF3-342},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:23238734},
pmc = {pmc:PMC6621731},
doi = {10.1523/JNEUROSCI.2681-12.2012},
url = {https://pub.dzne.de/record/136732},
}