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@ARTICLE{Abdeen:633348,
author = {Abdeen, Amr H. and Trist, Benjamin G. and Nikseresht, Sara
and Harwood, Richard and Roudeau, Stéphane and Rowlands,
Benjamin D. and Kreilaus, Fabian and Cottam, Veronica and
Mor, David and Richardson, Miriam and Siciliano, Joel and
Forkgen, Julia and Schaffer, Greta and Genoud, Sian and Li,
Anne A. and Proschogo, Nicholas and Antonio, Bernadeth and
Falkenberg, Gerald and Brueckner, Dennis and Kysenius, Kai
and Liddell, Jeffrey R. and Fat, Sandrine Chan Moi and Wu,
Sharlynn and Fifita, Jennifer and Lockwood, Thomas E. and
Bishop, David P. and Blair, Ian and Ortega, Richard and
Crouch, Peter J. and Double, Kay},
title = {{P}arkinson-like wild-type superoxide dismutase 1 pathology
induces nigral dopamine neuron degeneration in a novel
murine model},
journal = {Acta neuropathologica},
volume = {149},
number = {1},
issn = {0001-6322},
address = {Heidelberg},
publisher = {Springer},
reportid = {PUBDB-2025-02394},
pages = {22},
year = {2025},
abstract = {Atypical wild-type superoxide dismutase 1 (SOD1) protein
misfolding and deposition occurs specifically within the
degenerating substantia nigra pars compacta (SNc) in
Parkinson disease. Mechanisms driving the formation of this
pathology and relationship with SNc dopamine neuron health
are yet to be fully understood. We applied proteomic mass
spectrometry and synchrotron-based biometal quantification
to post-mortem brain tissues from the SNc of Parkinson
disease patients and age-matched controls to uncover key
factors underlying the formation of wild-type SOD1 pathology
in this disorder. We also engineered two of these factors -
brain copper deficiency and upregulated SOD1 protein levels
- into a novel mouse strain, termed the SOCK mouse, to
verify their involvement in the development of
Parkinson-like wild-type SOD1 pathology and their impact on
dopamine neuron health. Soluble SOD1 protein in the
degenerating Parkinson disease SNc exhibited altered
post-translational modifications, which may underlie changes
to the enzymatic activity and aggregation of the protein in
this region. These include decreased copper binding,
dysregulation of physiological glycosylation, and atypical
oxidation and glycation of key SOD1 amino acid residues. We
demonstrated that the biochemical profile introduced in SOCK
mice promotes the same post-translational modifications and
the development of Parkinson-like wild-type SOD1 pathology
in the midbrain and cortex. This pathology accumulates
progressively with age and is accompanied by nigrostriatal
degeneration and dysfunction, which occur in the absence of
α-synuclein deposition. These mice do not exhibit weight
loss nor spinal cord motor neuron degeneration,
distinguishing them from transgenic mutant SOD1 mouse
models. This study provides the first in vivo evidence that
mismetallation and altered post-translational modifications
precipitates wild-type SOD1 misfolding, dysfunction, and
deposition in the Parkinson disease brain, which may
contribute to SNc dopamine neuron degeneration. Our data
position this pathology as a novel drug target for this
disorder, with a particular focus on therapies capable of
correcting alterations to SOD1 post-translational
modifications.},
cin = {DOOR ; HAS-User / FS-PET-S},
ddc = {610},
cid = {I:(DE-H253)HAS-User-20120731 /
I:(DE-H253)FS-PET-S-20190712},
pnm = {633 - Life Sciences – Building Blocks of Life: Structure
and Function (POF4-633) / 6G3 - PETRA III (DESY) (POF4-6G3)
/ FS-Proposal: I-20230761 EC (I-20230761-EC)},
pid = {G:(DE-HGF)POF4-633 / G:(DE-HGF)POF4-6G3 /
G:(DE-H253)I-20230761-EC},
experiment = {EXP:(DE-H253)P-P06-20150101},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40042537},
doi = {10.1007/s00401-025-02859-6},
url = {https://bib-pubdb1.desy.de/record/633348},
}