MétaCan
Menu
Back to cohort
Record W4406237169 · doi:10.1126/sciimmunol.adq1697

Multiomics dissection of human RAG deficiency reveals distinctive patterns of immune dysregulation but a common inflammatory signature

2025· article· en· W4406237169 on OpenAlexaff
Marita Bosticardo, Kerry Dobbs, Ottavia M. Delmonte, Andrew J. Martins, Francesca Pala, Tomoki Kawai, Heather Kenney, Gloria Magro, Lindsey B. Rosen, Yasuhiro Yamazaki, Hsin‐Hui Yu, Enrica Calzoni, Yu Nee Lee, Can Liu, Jennifer Stoddard, Julie E. Niemela, Danielle Fink, Riccardo Castagnoli, Meredith Ramba, Aristine Cheng, Deanna Riley, Vasileios Oikonomou, Elana Shaw, Brahim Belaid, Sevgi Keleş, Waleed Al–Herz, Caterina Cancrini, Cristina Cifaldi, Safa Barış, Svetlana Sharapova, Catharina Schuetz, Andrew R. Gennery, Alexandra F. Freeman, Raz Somech, Sharon Choo, Silvia Giliani, Tayfun Güngör, Daniel Drozdov, Isabelle Meyts, Despina Moshous, Bénédicte Neven, Roshini S. Abraham, Aisha Elmarsafy, Maria Kanariou, Alejandra King, Francesco Licciardi, Mario Ernesto Cruz‐Muñoz, M. Cecilia Poli, Mehdi Adeli, Mattia Algeri, Fayhan Alroqi, Paul Bastard, Jenna Bergerson, Claire Booth, Ana Brett, Siobhan O. Burns, Manish J. Butte, Nurcicek Padem, Maite de la Morena, Ghassan Dbaibo, Suk See De Ravin, Dimana Dimitrova, Réda Djidjik, Mayra de Barros Dorna, Cullen M. Dutmer, Reem Elfeky, Fabio Facchetti, Ramsay Fuleihan, Luis Ignacio González‐Granado, Liis Haljasmägi, Hanadys Ale, Anthony Hayward, Anna Hilfanova, Winnie Ip, Blanka Kaplan, Neena Kapoor, Elif Karakoç-Aydıner, Jaanika Kärner, Michael D. Keller, Blachy J. Dávila Saldaña, Ayça Kıykım, Taco W. Kuijpers, Elena Kuznetsova, Elena A. Latysheva, Jennifer W. Leiding, Franco Locatelli, Guisela Alva‐Lozada, Christine McCusker, Fatih Çelmeli, Megan Morsheimer, Ahmet Özen, Nima Parvaneh, Srdjan Pašić, Alessandro Plebani, Kahn Preece, Susan E. Prockop, Inga Sakovich, Elena E. Starkova, Troy R. Torgerson, James Verbsky, Jolán E. Walter, Elizabeth Wisner, Deborah Draper, Katherine Myint‐Hpu, Michail S. Lionakis, Morgan Similuk, Magdalena Walkiewicz, Amy D. Klion, Steven M. Holland, Cihan Oguz, Dusan Bogunovic, Kai Kisand, Helen C. Su, John S. Tsang, Douglas B. Kuhns, Anna Villa, Sergio D. Rosenzweig, Stefania Pittaluga, Luigi D. Notarangelo, Rajarshi Ghosh, Bryce Siefert, Mari Tokita, Yan Jia, Colleen Jodarski, Mike Kamen, Nadjalisse Reynolds‐Lallement, Katie Lewis, Sarah A. Bannon, Adrienne Borges, Nicole Gentile

Bibliographic record

VenueScience Immunology · 2025
Typearticle
Languageen
FieldImmunology and Microbiology
TopicImmune Cell Function and Interaction
Canadian institutionsMcGill University Health CentreMontreal Children's Hospital
FundersNHLBI Division of Intramural ResearchNational Institute of Allergy and Infectious DiseasesSwedish Orphan BiovitrumNational Institutes of HealthGrifolsEesti TeadusagentuurLes Laboratories Pierre FabreAtara BiotherapeuticsDivision of Intramural Research, National Institute of Allergy and Infectious DiseasesMemorial Sloan-Kettering Cancer CenterBill and Melinda Gates FoundationAmgenRegeneron Pharmaceuticals
KeywordsImmune dysregulationImmune systemBiologyInflammationImmunology

Abstract

fetched live from OpenAlex

Human recombination-activating gene (RAG) deficiency can manifest with distinct clinical and immunological phenotypes. By applying a multiomics approach to a large group of RAG -mutated patients, we aimed at characterizing the immunopathology associated with each phenotype. Although defective T and B cell development is common to all phenotypes, patients with hypomorphic RAG variants can generate T and B cells with signatures of immune dysregulation and produce autoantibodies to a broad range of self-antigens, including type I interferons. T helper 2 (T H 2) cell skewing and a prominent inflammatory signature characterize Omenn syndrome, whereas more hypomorphic forms of RAG deficiency are associated with a type 1 immune profile both in blood and tissues. We used cellular indexing of transcriptomes and epitopes by sequencing (CITE-seq) analysis to define the cell lineage–specific contribution to the immunopathology of the distinct RAG phenotypes. These insights may help improve the diagnosis and clinical management of the various forms of the disease.

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)0.001
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: Bench or experimental
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.357
Threshold uncertainty score0.732

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0010.001
Science and technology studies0.0000.001
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 head0.006
GPT teacher head0.258
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.

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

Citations16
Published2025
Admission routes1
Has abstractyes

Explore more

Same venueScience ImmunologySame topicImmune Cell Function and InteractionFrench-language works237,207