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Record W4400874075 · doi:10.1101/2024.07.22.24310683

Adaptor protein 2 sigma subunit ( <i>AP2S1</i> ) variants associated with neurodevelopmental disorders

2024· preprint· en· W4400874075 on OpenAlexaff
Mark Stevenson, Asha L. Bayliss, Victoria Stokes, Katherine English, Kreepa Kooblall, Roman Fischer, Raphael Heilig, Iolanda Vendrell, Maria E. W. A. Albers, Meghan N. Bartos, Amber Begtrup, Alexia Bourgois, Rebecca Buchert, David J. Carey, Deanna Alexis Carere, Amanda Carnevale, Kristl G. Claeys, Benjamin Cogné, Gregory Costain, Nicole de Leeuw, Anne‐Sophie Denommé‐Pichon, Elizabeth Donner, Eftychia Drogouti, David A. Dyment, Balram Gangaram, Tobias B. Haack, Jeremy S. Haley, Solveig Heide, Ralf A. Husain, Bertrand Isidor, Louise Izatt, Adeline Jacquinet, Jane Juusola, Juliette J. Kahle, Boris Keren, Eric W. Klee, Evgenia Kokosali, Brendan C. Lanpher, Erica L. Macke, Elysa J. Marco, Kirsty McWalter, Bryce A. Mendelsohn, Aubrey Milunsky, Matthew Osmond, Amélie Piton, Angelika Rieß, Valentin Ruault, Patrick Rump, Sarah Schuhmann, Amelle L. Shillington, Diane T. Smelser, Lot Snijders Blok, Frédéric Tran Mau‐Them, Christos Tsakalidis, Abigail Turnwald, Koen L.I. van Gassen, Kristof Van Schil, Georgia Vasileiou, Marissa Vawter‐Lee, Marjolaine Willems, Marjolein H. Willemsen, Lily C. Wong‐Kisiel, Antje Wonneberger, Ioannis Zaganas, Fadil Hannan, Kate E Lines, Rajesh V. Thakker

Bibliographic record

VenuemedRxiv · 2024
Typepreprint
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGenetics and Neurodevelopmental Disorders
Canadian institutionsChildren's Hospital of Eastern OntarioSickKids FoundationUniversity of TorontoHospital for Sick Children
FundersMedical Research CouncilNational Institute for Health and Care ResearchRegeneron PharmaceuticalsCancer Research UKWellcome TrustDepartment of Health and Social CareCSL Behring
KeywordsProtein subunitSignal transducing adaptor proteinSigmaGeneticsComputational biologyBiologyMedicineGenePhysics

Abstract

fetched live from OpenAlex

Abstract Adaptor-Related Protein Complex 2 Sigma-1 Subunit ( AP2S1 ) encodes AP2σ2, which forms part of the heterotetrameric AP2 complex that is composed of α, β2, μ2, and σ2 subunits and has a pivotal role in clathrin-mediated endocytosis (CME) 1–3 . AP2S1 variants involving the Arg15 residue are associated with familial hypocalciuric hypercalcaemia type 3 (FHH3) 1,4–6 . Here, we report 5 different AP2S1 variants (AP2σ2: p.Arg10Trp, p.Arg10Gln, p.Lys18Glu, p.Lys18Asn and p.Arg61His) in 26 patients with neurodevelopmental delay, of whom >70% had epilepsy, 50% had brain abnormalities, and none had hypercalcaemia. All 5 variants decreased cell viability, 4 reduced CME transferrin uptake, and 4 disrupted interactions with other AP2 complex subunits, thereby affecting AP2 formation. Furthermore, AP2σ2 p.Arg10Trp had reduced interactions with 44 human proteins including intersectin 1, a component required for clathrin-coated pit formation and synaptic vesicle dynamics in neurones. Thus, our results show that AP2σ2 variants may disrupt CME and be associated with neurodevelopmental disorders.

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)0.001
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.003
Threshold uncertainty score0.011

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0000.001
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0010.001
Science and technology studies0.0010.001
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0010.000
Insufficient payload (model declined to judge)0.0030.000

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 head0.009
GPT teacher head0.211
Teacher spread0.202 · 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 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

Citations1
Published2024
Admission routes1
Has abstractyes

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