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Record W4414655438 · doi:10.1016/j.ajhg.2025.09.008

A cardiovascular, craniofacial, and neurodevelopmental disorder caused by loss-of-function variants in the eIF3 complex component genes EIF3A and EIF3B

2025· article· en· W4414655438 on OpenAlexafffund
Esra Erkut, Cherith Somerville, M. Schwartz, Laura A. McDonald, Qiliang Ding, Olivia Moran, Xin Chen, Roozbeh Manshaei, Anne-Sophie Riedijk, Marie-Therese Schnürer, Daniel C. Koboldt, Stylianos E. Antonarakis, Emma Bedoukian, Xavier Blanc, Laura K. Conlin, Helen Cox, Karin E. M. Diderich, Bri Dingmann, Christèle Dubourg, Frances Elmslie, Luis Fernando González Escobar, Rachel Gosselin, María J. Guillen Sacoto, C Haag, Lisa Herzig, Ramanand Jeeneea, Priti Kenia, Konstantinos Kolokotronis, Anna M. Kopps, Hayley Lees, Jacqueline Leonard, Jonathan Levy, Rebecca O. Littlejohn, Scott McLean, Nikhil Pattani, Laurence Perrin, Véronique Pingault, Chloé Quēlin, Emmanuelle Ranza, Anita Rauch, Sara L. Reichert, Joana Rosmaninho‐Salgado, Cara Skraban, Sérgio Sousa, Melissa Stuebben, Paolo Zanoni, Raymond H. Kim, Ian C. Scott, Rebekah Jobling

Bibliographic record

VenueThe American Journal of Human Genetics · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicRNA regulation and disease
Canadian institutionsUniversity of CalgarySickKids FoundationTed Rogers Centre for Heart ResearchHospital for Sick Children
FundersMedical Research CouncilUniversität ZürichAzrieli FoundationCanadian Institutes of Health ResearchNational Institute for Health and Care ResearchHeart of England NHS Foundation TrustCancer Research UKWellcome Trust
KeywordsCraniofacialZebrafishGenePhenotypeRibosomal RNAMutationGenomeCandidate gene

Abstract

fetched live from OpenAlex

Syndromic cardiac malformations can result in morbidity, yet their genetic etiology is only understood for a subset of individuals. Genome sequencing efforts in congenital anomaly cohorts may identify disease-associated variants in previously unrecognized genes. Through international matchmaking efforts, we identified eighteen individuals in total with de novo or loss-of-function variants in EIF3A (n = 4) or EIF3B (n = 14). The clinical phenotype varied but predominantly included cardiac defects, craniofacial dysmorphisms, mild developmental delays, and behavioral abnormalities. These genes encode core subunits of the eukaryotic initiation factor 3 (eIF3) complex, which plays a critical role in binding mRNA transcripts to the 40S ribosomal subunit during translation initiation. Both genes are highly constrained against loss of function, and animal models have demonstrated that disruptions in the eIF3 complex result in a range of developmental defects, including cardiovascular malformations. Additionally, EIF3B is located within the minimally overlapping region implicated in cardiac anomalies associated with 7p22.3 microdeletions. We sought to further study the role of these genes in syndromic congenital heart disease. To explore their functional impact, we generated zebrafish models with mutations in the orthologous eif3s10 and eif3ba genes, which resulted in developmental abnormalities, including thin heart tubes, lack of craniofacial cartilage, and embryonic lethality. We propose that pathogenic variants in EIF3A, as well as pathogenic variants or microdeletions involving EIF3B, cause a distinct autosomal-dominant neurodevelopmental syndrome characterized by cardiovascular and craniofacial manifestations.

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: Observational · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.612
Threshold uncertainty score0.291

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 head0.012
GPT teacher head0.255
Teacher spread0.243 · 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 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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