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@ARTICLE{Lange:285045,
author = {Lange, Lara M and Fang, Zih-Hua and Screven, Laurel and
Tan, Ai Huey and Alcalay, Roy N and Amouri, Rim and Bovenzi,
Roberta and Fenn, Matilda and Frost, Joshua L I and
Jankovic, Joseph and Jasaityte, Simona and Jaunmuktane, Zane
and Jeon, Beomseok and Sarmiento, Ignacio Juan Keller and
Krüger, Rejko and Kuhlenbäumer, Gregor and Lin, Chin-Hsien
and Pavelka, Lukas and Periñan, Maria Teresa and Sassi,
Samia Ben and Schirinzi, Tommaso and Shin, Jung Hwan and
Shulman, Joshua M and Tay, Yi Wen and Uitti, Ryan and
Warner, Tom and Wszolek, Zbigniew K and Wu, Lesley and Wu,
Ruey-Meei and Zeuner, Kirsten E and Blauwendraat, Cornelis
and Singleton, Andrew and Mencacci, Niccolò E and Morris,
Huw R and Lim, Shen-Yang and Lohmann, Katja and Klein,
Christine},
collaboration = {Program, Global Parkinson's Genetics},
title = {{R}are but {R}elevant? {A}ssessing {V}ariants in
{D}ystonia-{L}inked {G}enes in {P}arkinson's {D}isease.},
journal = {Movement disorders},
volume = {41},
number = {1},
issn = {0885-3185},
address = {New York, NY},
publisher = {Wiley},
reportid = {DZNE-2026-00169},
pages = {247 - 259},
year = {2026},
abstract = {Dystonia and Parkinson's disease (PD) exhibit clinical and
genetic overlap, but the relevance of dystonia gene variants
in PD remains unclear.The aim was to assess the frequency of
dystonia-linked pathogenic variants in PD.We screened
sequencing data from 15,684 individuals (8272 PD, 3200
atypical parkinsonism, and 4212 unaffected) from the Global
Parkinson's Genetics Program (GP2) and Accelerating
Medicines Partnership-Parkinson's Disease (AMP-PD) for
variants in genes linked to isolated dystonia,
dystonia-parkinsonism, and myoclonus-dystonia.Pathogenic
variants were identified only in PD patients. Forty-five PD
individuals $(0.54\%)$ carried 26 distinct (likely)
pathogenic variants in nine dystonia-linked genes, most
frequently in GCH1, followed by VPS16.Though rare,
pathogenic variants in dystonia-linked genes are present in
clinically and pathologically diagnosed PD. Our results
reinforce GCH1 as a PD-relevant gene with clinical
implications, whereas variants identified in other genes are
rare and of uncertain relation to the PD phenotype. © 2025
The Author(s). Movement Disorders published by Wiley
Periodicals LLC on behalf of International Parkinson and
Movement Disorder Society.},
keywords = {Humans / Parkinson Disease: genetics / Parkinson Disease:
complications / Male / Female / Middle Aged / Dystonia:
genetics / Aged / GTP Cyclohydrolase: genetics / Dystonic
Disorders: genetics / Adult / Vesicular Transport Proteins:
genetics / GCH1 (Other) / Parkinson's disease (Other) /
VPS16 (Other) / dystonia (Other) / monogenic (Other) / GTP
Cyclohydrolase (NLM Chemicals) / GCH1 protein, human (NLM
Chemicals) / Vesicular Transport Proteins (NLM Chemicals)},
cin = {AG Heutink},
ddc = {610},
cid = {I:(DE-2719)1210002},
pnm = {354 - Disease Prevention and Healthy Aging (POF4-354)},
pid = {G:(DE-HGF)POF4-354},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41074695},
pmc = {pmc:PMC12882037},
doi = {10.1002/mds.70073},
url = {https://pub.dzne.de/record/285045},
}