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Record W4415525656 · doi:10.1101/2025.10.17.25337589

RBMX functional retrocopy safeguards brain development

2025· preprint· en· W4415525656 on OpenAlexaff
Pierre Tilliole, Carolin Mattausch, Peggy Tilly, Elsa Leitão, Lucile Boutaud, Daphné Lehalle, Isabelle An, Emanuela Argilli, Sharon Aufox, Bert Callewaert, Perrine Charles, Jessica Cinkornpumin, Thomas Courtin, Marco Dalla Vecchia, Erica E. Davis, Boyan Dimitrov, William B. Dobyns, Ekaterina Epifanova, Erwan Grandgirard, Matthieu Jung, Sarah Jurgensmeyer, Sabine Kaya, Boris Keren, Tahir Naeem Khan, Élodie Lejeune, Mingfeng Li, Yannick Marie, Bastien Morlet, Caroline Nava, William A. Pastor, Damien Plassard, Carlos E. Prada, Agnès Rastetter, No eacutemie Schwaller, Nenad Šestan, Elliott H. Sherr, Suzanna L. Temple, Jude-Felix Tenywa, Sylvia Tielens, Arie van Haeringen, Helen Whitley, Laurent Nguyen, Muriel Rhinn, Stephan C. Collins, Valérie Cormier‐Daire, Tania Attié‐Bitach, Binnaz Yalcin, Christel Depienne, Juliette D. Godin

Bibliographic record

VenuemedRxiv · 2025
Typepreprint
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicCongenital heart defects research
Canadian institutionsMcGill University
Fundersnot available
KeywordsPhenotypeBrain developmentGeneRNA splicingHuman brainAlternative splicingRNATranscriptome

Abstract

fetched live from OpenAlex

ABSTRACT Retrotransposition has generated thousands of intronless gene copies in mammalian genomes, yet their contribution to brain development and evolution remains largely unexplored. Here we uncover a critical role for RBMX retrocopy in shaping neurodevelopment and modulating disease. Pathogenic variants in RBMX , an X-linked splicing regulator, cause intellectual disability, microcephaly, and cortical malformations. Through integrated human genetic, cellular, and mouse model studies, we show that RBMX pathogenic variants disrupt cortical development through both loss- and gain-of-function mechanisms. Surprisingly, despite severe phenotypes in humans, Rbmx -deficient mice display only mild cortical abnormalities, a discrepancy likely due to compensation by Rbmxl1 , a retrocopy that arose independently in mice and humans. We demonstrate that RBMX and RBMXL1 share protein and RNA partners and act redundantly in brain development, with RBMXL1 buffering the impact of RBMX deficiency. These findings establish retrocopies as functional paralogs safeguarding neurodevelopment, and suggest that functional RBMX retrocopy could contribute to the robustness and evolutionary diversification of mammalian brain.

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 machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: Bench or experimental
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.003
Threshold uncertainty score0.009

Distilled classifier scores by category (both heads)

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 communication0.0010.000
Open science0.0000.001
Research integrity0.0000.001
Insufficient payload (model declined to judge)0.0030.001

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 head0.025
GPT teacher head0.310
Teacher spread0.285 · 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 source (direct Gemma or distilled Codex), 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

Citations0
Published2025
Admission routes1
Has abstractyes

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