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@ARTICLE{Robustelli:163399,
author = {Robustelli, Paul and Ibanez de Opakua, Alain and
Campbell-Bezat, Cecily and Giordanetto, Fabrizio and Becker,
Stefan and Zweckstetter, Markus and Pan, Albert C and Shaw,
David E},
title = {{M}olecular {B}asis of {S}mall-{M}olecule {B}inding to
α-{S}ynuclein.},
journal = {Journal of the American Chemical Society},
volume = {144},
number = {6},
issn = {1520-5126},
address = {Washington, DC},
publisher = {American Chemical Society},
reportid = {DZNE-2022-00161},
pages = {2501 - 2510},
year = {2022},
note = {(CC BY-NC-ND)},
abstract = {Intrinsically disordered proteins (IDPs) are implicated in
many human diseases. They have generally not been amenable
to conventional structure-based drug design, however,
because their intrinsic conformational variability has
precluded an atomic-level understanding of their binding to
small molecules. Here we present long-time-scale,
atomic-level molecular dynamics (MD) simulations of
monomeric α-synuclein (an IDP whose aggregation is
associated with Parkinson's disease) binding the
small-molecule drug fasudil in which the observed
protein-ligand interactions were found to be in good
agreement with previously reported NMR chemical shift data.
In our simulations, fasudil, when bound, favored certain
charge-charge and π-stacking interactions near the C
terminus of α-synuclein but tended not to form these
interactions simultaneously, rather breaking one of these
interactions and forming another nearby (a mechanism we term
dynamic shuttling). Further simulations with small molecules
chosen to modify these interactions yielded binding
affinities and key structural features of binding consistent
with subsequent NMR experiments, suggesting the potential
for MD-based strategies to facilitate the rational design of
small molecules that bind with disordered proteins.},
keywords = {1-(5-Isoquinolinesulfonyl)-2-Methylpiperazine: analogs $\&$
derivatives / 1-(5-Isoquinolinesulfonyl)-2-Methylpiperazine:
chemistry / 1-(5-Isoquinolinesulfonyl)-2-Methylpiperazine:
metabolism / Amino Acid Sequence / Hydrogen Bonding /
Intrinsically Disordered Proteins: chemistry / Intrinsically
Disordered Proteins: metabolism / Ligands / Molecular
Conformation / Molecular Dynamics Simulation / Protein
Binding / Small Molecule Libraries: chemistry / Small
Molecule Libraries: metabolism / alpha-Synuclein: metabolism
/ Intrinsically Disordered Proteins (NLM Chemicals) /
Ligands (NLM Chemicals) / Small Molecule Libraries (NLM
Chemicals) / alpha-Synuclein (NLM Chemicals) /
1-(5-Isoquinolinesulfonyl)-2-Methylpiperazine (NLM
Chemicals) / fasudil (NLM Chemicals)},
cin = {AG Zweckstetter},
ddc = {540},
cid = {I:(DE-2719)1410001},
pnm = {352 - Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-352},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:35130691},
pmc = {pmc:PMC8855421},
doi = {10.1021/jacs.1c07591},
url = {https://pub.dzne.de/record/163399},
}