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@ARTICLE{Andersen:282580,
author = {Andersen, Olav M and de Waal, Matthijs W J and Monti,
Giulia and Tesi, Niccolo and Jensen, Anne Mette G and de
Geus, Christa and van Spaendonk, Rosalina and Vogel, Maartje
and Ahmad, Shahzad and Amin, Najaf and Amouyel, Philippe and
Beecham, Gary W and Bellenguez, Céline and Berr, Claudine
and Bis, Joshua C and Boland, Anne and Bossù, Paola and
Bouwman, Femke and Bras, Jose and Charbonnier, Camille and
Clarimon, Jordi and Cruchaga, Carlos and Daniele, Antonio
and Dartigues, Jean-François and Debette, Stéphanie and
Deleuze, Jean-François and Denning, Nicola and DeStefano,
Anita L and Dols-Icardo, Oriol and van Duijn, Cornelia M and
Farrer, Lindsay A and Fernández, Maria Victoria and van der
Flier, Wiesje M and Fox, Nick C and Galimberti, Daniela and
Genin, Emmanuelle and Gille, Johan J P and Grenier-Boley,
Benjamin and Grozeva, Detelina and Guen, Yann Le and
Guerreiro, Rita and Haines, Jonathan L and Holmes, Clive and
Hummerich, Holger and Arfan Ikram, M. and Kamran Ikram, M.
and Kawalia, Amit and Kraaij, Robert and Lambert,
Jean-Charles and Lathrop, Marc and Lemstra, Afina W and
Lleó, Alberto and Myers, Richard M and Mannens, Marcel M A
M and Marshall, Rachel and Martin, Eden R and Masullo, Carlo
and Mayeux, Richard and Mead, Simon and Mecocci, Patrizia
and Meggy, Alun and Mol, Merel O and Nacmias, Benedetta and
Naj, Adam C and Napolioni, Valerio and Nicholas Cochran, J.
and Nicolas, Gaël and Pasquier, Florence and Pastor, Pau
and Pericak-Vance, Margaret A and Pijnenburg, Yolande A L
and Piras, Fabrizio and Quenez, Olivier and Ramirez, Alfredo
and Raybould, Rachel and Redon, Richard and Reinders, Marcel
J T and Richard, Anne-Claire and Riedel-Heller, Steffi G and
Rivadeneira, Fernando and van Rooij, Jeroen G J and
Rousseau, Stéphane and Ryan, Natalie S and Sanchez-Juan,
Pascual and Schellenberg, Gerard D and Scheltens, Philip and
Schott, Jonathan M and Seshadri, Sudha and Sie, Daoud and
Sims, Rebecca and Sistermans, Erik A and Sorbi, Sandro and
van Swieten, John C and Tijms, Betty and Uitterlinden,
André G and Visser, Pieter Jelle and Wagner, Michael and
Wallon, David and Wang, Li-San and Williams, Julie and
Yokoyama, Jennifer S and Zarea, Aline and van der Lee, Sven
J and Olsen, Johan G and Hulsman, Marc and Holstege, Henne},
title = {{D}omain mapping of disease mutations reveals pathogenic
{SORL}1 variants in {A}lzheimer's disease.},
journal = {Molecular neurodegeneration},
volume = {20},
number = {1},
issn = {1750-1326},
address = {London},
publisher = {Biomed Central},
reportid = {DZNE-2025-01340},
pages = {122},
year = {2025},
abstract = {Protein truncating variants (PTVs) in SORL1 are observed
almost exclusively in Alzheimer’s Disease (AD) cases, but
the effect of rare SORL1 missense variants is unclear.To
identify high-priority missense variants (HPVs), we applied
‘domain mapping of disease mutations’ for the 637 unique
coding SORL1 variants detected in 18,959 AD-cases and 21,893
non-demented controls.In this sample, PTVs and HPVs
associated with respectively a 35- and 10-fold increased
risk of early onset AD and 17- and 6-fold increased risk of
overall AD. The median age at onset (AAO) of PTV- and
HPV-carriers was 62 and 64 years, and APOE-genotype
contributed to AAO-variability. The median AAO of PTV- and
HPV-carriers is ~8–10 years earlier than wild-type SORL1
carriers, matched for APOE-genotype. Specific HPVs are
highly penetrant and lead to earlier AAOs than PTVs,
suggesting possible dominant negative effects.Our results
justify a debate on whether HPV carriers should be
considered for clinical counseling.The online version
contains supplementary material available at
10.1186/s13024-025-00907-z.},
keywords = {Age at onset (Other) / Alzforum mutation database (Other) /
Alzheimer’s disease (Other) / Disease risk (Other) /
Domain-mapping disease-mutations (Other) / Genetics (Other)
/ Penetrance (Other) / Rare variants (Other) / SORL1 (Other)
/ SORLA (Other)},
cin = {AG Wagner / Patient Studies (Bonn)},
ddc = {570},
cid = {I:(DE-2719)1011201 / 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:41327266},
pmc = {pmc:PMC12667055},
doi = {10.1186/s13024-025-00907-z},
url = {https://pub.dzne.de/record/282580},
}