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Record W4416599612 · doi:10.1038/s41588-025-02400-1

Proteome-wide model for human disease genetics

2025· article· en· W4416599612 on OpenAlexaff
Rose Orenbuch, Courtney A. Shearer, Aaron W. Kollasch, Thomas A. Hopf, Lood van Niekerk, Dinko Franceschi, Mafalda Dias, Jonathan Frazer, Debora S. Marks

Bibliographic record

VenueNature Genetics · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGenomics and Rare Diseases
Canadian institutionsArtificial Intelligence in Medicine (Canada)
FundersNational Cancer InstituteNational Institutes of HealthNational Science FoundationGeneralitat de CatalunyaAgencia Estatal de InvestigaciónInvitaeMinisterio de Ciencia e InnovaciónCentres de Recerca de Catalunya
KeywordsDiseaseMissense mutationLimitingPopulationHuman diseaseHuman genetic variationGenerative grammarMedical geneticsHuman genetics

Abstract

fetched live from OpenAlex

Missense variants remain a challenge in genetic interpretation owing to their subtle and context-dependent effects. Although current prediction models perform well in known disease genes, their scores are not calibrated across the proteome, limiting generalizability. To address this knowledge gap, we developed popEVE, a deep generative model combining evolutionary and human population data to estimate variant deleteriousness on a proteome-wide scale. popEVE achieves state-of-the-art performance without overestimating the burden of deleterious variants and identifies variants in 442 genes in a severe developmental disorder cohort, including 123 novel candidates. These genes are functionally similar to known disease genes, and their variants often localize to critical regions. Remarkably, popEVE can prioritize likely causal variants using only child exomes, enabling diagnosis even without parental sequencing. This work provides a generalizable framework for rare disease variant interpretation, especially in singleton cases, and demonstrates the utility of calibrated, evolution-informed scoring models for clinical genomics.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

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)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.386
Threshold uncertainty score0.919

Codex and Gemma teacher scores by category

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

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 head0.279
Teacher spread0.272 · 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.

The models applied no category: nothing in the taxonomy fit this work.
Study designBench or experimental
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

Citations18
Published2025
Admission routes1
Has abstractyes

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