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@ARTICLE{FloresFernandez:1032082,
author = {Flores-Fernandez, Jose Miguel and Pesch, Verena and
Sriraman, Aishwarya and Chimal-Juarez, Enrique and Amidian,
Sara and Wang, Xiongyao and Duckering, Caleb and Fang,
Andrew and Reithofer, Sara and Ma, Liang and Cortez,
Leonardo M. and Sim, Valerie L. and Tamgüney, Gültekin and
Wille, Holger},
title = {{R}ational design of structure‐based vaccines targeting
misfolded alpha‐synuclein conformers of {P}arkinson's
disease and related disorders},
journal = {Bioengineering $\&$ translational medicine},
volume = {9},
number = {4},
issn = {2380-6761},
address = {Hoboken, NJ},
publisher = {Wiley},
reportid = {FZJ-2024-05985},
pages = {e10665},
year = {2024},
abstract = {Synucleinopathies, including Parkinson's disease (PD),
multiple system atrophy (MSA), and dementia with Lewy bodies
(DLB), are neurodegenerative disorders caused by the
accumulation of misfolded alpha-synuclein protein.
Developing effective vaccines against synucleinopathies is
challenging due to the difficulty of stimulating an
immune-specific response against alpha-synuclein without
causing harmful autoimmune reactions, selectively targeting
only pathological forms of alpha-synuclein. Previous
attempts using linear peptides and epitopes without control
of the antigen structure failed in clinical trials. The
immune system was unable to distinguish between native
alpha-synuclein and its amyloid form. The prion domain of
the fungal HET-s protein was selected as a scaffold to
introduce select epitopes from the surface of
alpha-synuclein fibrils. Four vaccine candidates were
generated by introducing specific amino acid substitutions
onto the surface of the scaffold protein. The approach
successfully mimicked the stacking of the parallel
in-register beta-sheet structure seen in alpha-synuclein
fibrils. All vaccine candidates induced substantial levels
of IgG antibodies that recognized pathological
alpha-synuclein fibrils derived from a synucleinopathy mouse
model. Furthermore, the antisera recognized pathological
alpha-synuclein aggregates in brain lysates from patients
who died from DLB, MSA, or PD, but did not recognize linear
alpha-synuclein peptides. Our approach, based on the
rational design of vaccines using the structure of
alpha-synuclein amyloid fibrils and strict control over the
exposed antigen structure used for immunization, as well as
the ability to mimic aggregated alpha-synuclein, provides a
promising avenue toward developing effective vaccines
against alpha-synuclein fibrils.},
cin = {IBI-7},
ddc = {600},
cid = {I:(DE-Juel1)IBI-7-20200312},
pnm = {5244 - Information Processing in Neuronal Networks
(POF4-524)},
pid = {G:(DE-HGF)POF4-5244},
typ = {PUB:(DE-HGF)16},
pubmed = {39036077},
UT = {WOS:001198609000001},
doi = {10.1002/btm2.10665},
url = {https://juser.fz-juelich.de/record/1032082},
}