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@ARTICLE{Pogoda:145091,
author = {Pogoda, Michaela and Hilke, Franz-Joachim and Lohmann, Ebba
and Sturm, Marc and Lenz, Florian and Matthes, Jakob and
Muyas, Francesc and Ossowski, Stephan and Hoischen,
Alexander and Faust, Ulrike and Sepahi, Ilnaz and Casadei,
Nicolas and Poths, Sven and Riess, Olaf and Schroeder,
Christopher and Grundmann, Kathrin},
title = {{S}ingle {M}olecule {M}olecular {I}nversion {P}robes for
{H}igh {T}hroughput {G}ermline {S}creenings in {D}ystonia.},
journal = {Frontiers in neurology},
volume = {10},
issn = {1664-2295},
address = {Lausanne},
publisher = {Frontiers Research Foundation},
reportid = {DZNE-2020-00450},
pages = {1332},
year = {2019},
abstract = {Background: This study's aim was to investigate a large
cohort of dystonia patients for pathogenic and rare variants
in the ATM gene, making use of a new, cost-efficient
enrichment technology for NGS-based screening. Methods:
Single molecule Molecular Inversion Probes (smMIPs) were
used for targeted enrichment and sequencing of all protein
coding exons and exon-intron boundaries of the ATM gene in
373 dystonia patients and six positive controls with known
ATM variants. Additionally, a rare-variant association study
was performed. Results: One patient $(0.3\%)$ was compound
heterozygous and 21 others were carriers of variants of
unknown significance (VUS) in the ATM gene. Although
mutations in sporadic dystonia patients are not common,
exclusion of pathogenic variants is crucial to recognize a
potential tumor predisposition syndrome. SmMIPs produced
similar results as routinely used NGS-based approaches.
Conclusion: Our results underline the importance of
implementing ATM in the routine genetic testing of dystonia
patients and confirm the reliability of smMIPs and their
usability for germline screenings in rare neurodegenerative
conditions.},
cin = {AG Gasser 1},
ddc = {610},
cid = {I:(DE-2719)1210000},
pnm = {345 - Population Studies and Genetics (POF3-345)},
pid = {G:(DE-HGF)POF3-345},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:31920950},
pmc = {pmc:PMC6930228},
doi = {10.3389/fneur.2019.01332},
url = {https://pub.dzne.de/record/145091},
}