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Record W4415450211 · doi:10.1016/j.ekir.2025.09.053

Exome-Wide Analysis Identifies a Rare EXD3 Missense Variant Associated With Diabetic Kidney Disease

2025· article· en· W4415450211 on OpenAlexafffund
Niina Sandholm, Joanne B. Cole, Viji Nair, Eoin Brennan, Elena Giardini, Jani K. Haukka, Eunji Ha, Anna Syreeni, Rany M. Salem, Damian Fermin, Josep V. Mercader, Laura J. Smyth, Claire Hill, Josyf C. Mychaleckyj, Stuart J. McGurnaghan, Rachel G. Miller, Tina Costacou, Barbara E.K. Klein, Janet K. Snell‐Bergeon, Andrew D. Paterson, Rasa Verkauskienė, Jeļizaveta Sokolovska, Nicolae Mircea Panduru, Gianpaolo Zerbini, Kerstin Brismar, Andrzej S. Krolewski, Valma Harjutsalo, Peter Rossing, Samy Hadjadj, Gareth J. McKay, Amy Jayne McKnight, Alexander P. Maxwell, Katalin Suszták, Catherine Godson, Matthias Kretzler, Joel N. Hirschhorn, José C. Florez, Per‐Henrik Groop

Bibliographic record

VenueKidney International Reports · 2025
Typearticle
Languageen
FieldMedicine
TopicRenal Diseases and Glomerulopathies
Canadian institutionsInstitute of Nutrition, Metabolism and DiabetesSickKids Foundation
FundersJDRFNational Institute of Diabetes and Digestive and Kidney DiseasesConsiliul National al Cercetarii StiintificeGeorge M. O'Brien Michigan Kidney Translational Core CenterNational Institutes of HealthSigrid Juséliuksen SäätiöMedical Research CouncilSamfundet FolkhälsanNovo NordiskGenesis HealthCare SystemNovo Nordisk FondenUniversity of VirginiaAcademy of FinlandDepartment for the EconomyUK Research and InnovationScience Foundation IrelandFolkhälsanin TutkimussäätiöJuvenile Diabetes Research Foundation CanadaWilhelm och Else Stockmanns StiftelseHelsingin ja Uudenmaan SairaanhoitopiiriUniversity of Pittsburgh
KeywordsMissense mutationDiseasePodocyteKidney diseaseDiabetic nephropathyRare disease

Abstract

fetched live from OpenAlex

Introduction Diabetic kidney disease (DKD) is a major complication of diabetes, with genetic factors contributing to its progression.While genome-wide association studies have identified common variants, the role of low-frequency and rare coding variants remains underexplored. MethodsWe performed exome-wide meta-analysis of up to 10,312 individuals with type 1 diabetes (T1D) genotyped using genome arrays with focused exome content.We included ten DKD definitions based on albuminuria, eGFR, or both.We analyzed non-synonymous variants individually, and using gene-level analyses for low-frequency (minor allele frequency <5%) and rare (<1%) variants.Replication was performed in 10,066 participants with T1D and in UK Biobank participants with type 2 diabetes.Gene expression was assessed in cultured human podocytes. ResultsIn addition to the known COL4A3 variant, a novel rare missense variant in EXD3 (p.Asp555Asn, rs200080727, MAF=0.4%) was associated with DKD (OR=8.7,p=4.510 -9 ).The variant was predicted to be deleterious and EXD3 was downregulated in DKD in kidney expression datasets.EXD3 knockdown in a cultured human podocyte cell line reduced nephrin gene expression, suggesting a functional role in podocyte biology.Gene-level analyses identified seven DKD-associated genes (p<3.410 -6 ), including MUC5B, which harbored multiple low-frequency missense variants and with evidence of replication.Replication in UK Biobank supported the association of EXD3 rs200080727 with albuminuria (p=0.014). ConclusionThis study identified a rare EXD3 variant with a strong effect on DKD risk in T1D.Functional data support a role for EXD3 in podocyte integrity and DKD pathogenesis.However, further functional investigations are necessary to understand the underlying molecular mechanisms.

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How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.012
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch, Insufficient payload (model declined to judge)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.131
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.012
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0010.001
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0010.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.007
GPT teacher head0.259
Teacher spread0.252 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

Study designObservational
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations2
Published2025
Admission routes2
Has abstractyes

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