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@ARTICLE{Peikert:277726,
author = {Peikert, Kevin and Spranger, Adrian and
Miltenberger-Miltenyi, Gabriel and Glaß, Hannes and
Falkenburger, Björn and Klose, Christian and Tyteca,
Donatienne and Hermann, Andreas},
title = {{P}hosphatidylethanolamines are the {M}ain {L}ipid {C}lass
{A}ltered in {R}ed {B}lood {C}ells from {P}atients with
{VPS}13{A} {D}isease/{C}horea-{A}canthocytosis.},
journal = {Movement disorders},
volume = {40},
number = {3},
issn = {0885-3185},
address = {New York, NY},
publisher = {Wiley},
reportid = {DZNE-2025-00447},
pages = {544 - 549},
year = {2025},
abstract = {VPS13A disease is an ultra-rare disorder caused by loss of
function mutations in VPS13A characterized by striatal
degeneration and by red blood cell (RBC) acanthocytosis.
VPS13A is a bridge-like protein mediating lipid transfer at
membrane contact sites.To assess the lipid composition of
patient-derived RBCs.RBCs collected from 5 VPS13A disease
patients and 12 control subjects were analyzed by mass
spectrometry (lipidomics).While we found no significant
differences in the overall lipid class level, alterations in
certain species were detected: phosphatidylethanolamine
species with both longer chain length and higher
unsaturation were increased in VPS13A disease samples.
Specific ceramide, phosphatidylcholine, and sphingomyelin
species were also altered.The presented alterations of
particular lipid species in RBCs in VPS13A disease may
contribute to (1) the understanding of acanthocyte
formation, and (2) future biomarker identification. Lipid
distribution seems to play a key role in the pathophysiology
of VPS13A disease. © 2024 The Author(s). Movement Disorders
published by Wiley Periodicals LLC on behalf of
International Parkinson and Movement Disorder Society.},
keywords = {Humans / Neuroacanthocytosis: genetics /
Neuroacanthocytosis: metabolism / Neuroacanthocytosis: blood
/ Neuroacanthocytosis: pathology /
Phosphatidylethanolamines: metabolism / Erythrocytes:
metabolism / Male / Vesicular Transport Proteins: genetics /
Vesicular Transport Proteins: metabolism / Female / Adult /
Middle Aged / Lipidomics: methods / Acanthocytes: metabolism
/ Acanthocytes: pathology / VPS13A protein, human (NLM
Chemicals) / Phosphatidylethanolamines (NLM Chemicals) /
Vesicular Transport Proteins (NLM Chemicals)},
cin = {AG Hermann / AG Falkenburger},
ddc = {610},
cid = {I:(DE-2719)1511100 / I:(DE-2719)1710012},
pnm = {353 - Clinical and Health Care Research (POF4-353)},
pid = {G:(DE-HGF)POF4-353},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39665525},
pmc = {pmc:PMC11926492},
doi = {10.1002/mds.30086},
url = {https://pub.dzne.de/record/277726},
}