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@ARTICLE{Grover:285361,
author = {Grover, Sandeep and Gockel, Ines and Latiano, Anna and
Mokrowiecka, Anna and Dasmeh, Pouria and Wouters, Mira M and
Vackova, Zuzana and Haas, Stephan L and Triantafyllou, Tania
and Kreuser, Nicole and Trautmann, Jessica and Niebisch,
Stefan and Hess, Timo and Thieme, Rene and Bigge, Jessica
and Louis, Hubert and Quertinmont, Eric and Meirhaeghe,
Aline and Muntaner, Manon and Amouyel, Philippe and
Gourcerol, Guillaume and Bruley des Varannes, Stanislas and
Mion, Francois and Vieth, Michael and Scarmeas, Nikolaos and
Palmieri, Orazio and Tavano, Francesca and De Giorgio,
Roberto and Galimberti, Daniela and Arighi, Andrea and
Arosio, Beatrice and Bruno, Marco and Wasielica-Berger,
Justyna and Gawron-Kiszka, Magdalena and Janiak, Maria and
Siepsiak, Magdalena and Adrych, Krystian and Marek, Tomasz
and Dabrowski, Andrzej and Majewski, Marek and Gietka, Piotr
and Gonciarz, Maciej and Pérez de la Serna, Julio and
Martínez, Laisy Zacarías and Giedraitis, Vilmantas and
Kilander, Lena and Fratiglioni, Laura and Real, Luis Miguel
and Spicak, Julius and Tack, Jan and Heilmann-Heimbach,
Stefanie and Nöthen, Markus and Ingelsson, Martin and
Graff, Caroline and Ruiz, Agustín and Lambert, Jean-Charles
and Ramirez, Alfredo and Eckardt, Alexander J and Müller,
Michaela and Knapp, Michael and Wissinowski, Thaddäus T and
Keller, Jutta and Bruns, Christiane Josephine and Gerges,
Christian and Neuhaus, Horst and Rösch, Thomas and
Siegmund, Britta and Schumacher, Brigitte and Venerito,
Marino and Ruiz de León, Antonio and Rosati, Riccardo and
Annese, Vito and Fumagalli, Uberto and Laghi, Luigi and
Urcelay, Elena and Vavasseur, Fabienne and Roman, Sabine and
Zhou, Pinghong and Li, Quanlin and Liu, Zuqiang and Rahden,
Burkhard H A von and Theodorou, Dimitris and
Malecka-Wojciesko, Ewa and Maj, Carlo and Vigo, Ana G and
Martinek, Jan and Boeckxstaens, Guy and Schumacher,
Johannes},
title = {{F}irst genome-wide association study reveals
immune-mediated aetiopathology in idiopathic achalasia.},
journal = {Gut},
volume = {75},
number = {3},
issn = {0017-5749},
address = {London},
publisher = {BMJ Publishing Group},
reportid = {DZNE-2026-00226},
pages = {476 - 485},
year = {2025},
abstract = {Idiopathic achalasia (IA) is characterised by the
degeneration of neurons in the myenteric plexus leading to
an irreversible impaired oesophageal function. Although
immune-mediated mechanisms have been proposed, the
underlying aetiopathology of IA remains poorly
understood.This study aimed to uncover the genetic risk
architecture of IA.We carried out the first genome-wide
association study (GWAS) on 4602 European patients with IA
and 10 766 ethnically-matched controls.A single nucleotide
polymorphism (SNP) in HLA-DQB1 leading to an 8-amino acid
insertion on the protein level conferred strongest IA risk
(PQGPPPAG: p=3.27×10-68, OR=2.45). Conditional analyses
within the HLA locus revealed a complex genetic risk
architecture. Three additional amino acid positions showed
independent IA association (Omnibus p<5×10-8). These refer
to positions 41 and 130 in HLA-DQα1, position 45 in
HLA-DQβ1 and position 86 in HLA-DRβ1. Together, these
findings highlight the pivotal role of class II HLA genetic
variation in IA pathogenesis. Outside HLA, three independent
variants showed IA association (p<5×10-8). One leads to an
amino acid substitution with functional effect in PTPN22.
Another risk variant leads to a downregulated expression of
TNFSF8, TNFSF15 and TNC in immune cells. The third risk SNP
is located near ZNF365, but the exact underlying cellular
mechanism remains unknown. Beyond the single marker level,
polygenic risk scores revealed that patients with IA can be
stratified based on their genetic risk. In addition, IA
shows a shared aetiopathology with Crohn's disease
(rg=0.335). Integrating GWAS and single-cell RNA-sequencing
data from the myenteric plexus showed that the memory T-cell
type FOS+Tc4+CD8+ plays a central role in IA development
(p=2.50×10-19).This GWAS led to the identification of SNPs,
cellular mechanisms and cell types that are involved in IA
aetiopathology.},
keywords = {ACHALASIA (Other) / GENETIC POLYMORPHISMS (Other) /
GENETICS (Other) / HLA CLASS II ALLELES (Other) / RNA
EXPRESSION (Other)},
cin = {Patient Studies (Bonn)},
ddc = {610},
cid = {I:(DE-2719)1011101},
pnm = {353 - Clinical and Health Care Research (POF4-353)},
pid = {G:(DE-HGF)POF4-353},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41136183},
doi = {10.1136/gutjnl-2024-334498},
url = {https://pub.dzne.de/record/285361},
}