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Record W2946995703 · doi:10.1073/pnas.1819825116

Gain-of-function mutations in a member of the Src family kinases cause autoinflammatory bone disease in mice and humans

2019· article· en· W2946995703 on OpenAlexaff
Koichiro Abe, Allison Cox, Nobuhiko Takamatsu, Gabriel Velez, Ronald M. Laxer, Shirley M. L. Tse, Vinit B. Mahajan, Alexander G. Bassuk, Helmut Fuchs, Polly J. Ferguson, Martin Hrabě de Angelis

Bibliographic record

VenueProceedings of the National Academy of Sciences · 2019
Typearticle
Languageen
FieldMedicine
TopicOsteomyelitis and Bone Disorders Research
Canadian institutionsHospital for Sick ChildrenUniversity of Toronto
FundersNational Institute of Arthritis and Musculoskeletal and Skin DiseasesNational Institute of General Medical SciencesNational Eye InstituteJapan Society for the Promotion of ScienceNational Institutes of HealthJapan Society for the Promotion of Science LondonNational Institute on AgingResearch to Prevent BlindnessINFRAFRONTIERBundesministerium für Bildung und Forschung
KeywordsMissense mutationBiologyGeneticsPhenotypePositional cloningMutationExome sequencingMutantLoss functionGeneCancer research

Abstract

fetched live from OpenAlex

Significance Chronic recurrent multifocal osteomyelitis (CRMO) is an autoinflammatory bone disease that presents with bone destruction occurring primarily in children. In a mouse ENU mutagenesis screen, the Ali18 strain was isolated because of spontaneous inflammation in the joints and bones. Sequencing candidate genes in the Ali18 critical region identified a missense mutation in the C-terminal regulatory region of the Src family kinase (SFK) member, Fgr . Genome editing revealed Fgr dependency of the inflammatory phenotype in Ali18 mice. Further, whole exome sequencing in our CRMO cohort identified two patients with missense mutations in FGR . In vitro functional assays confirm altered protein function. This work identifies FGR as a CRMO susceptibility gene and suggests that targeting SFKs may be useful in its treatment.

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 score0.010

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0010.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0010.001
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 head0.047
GPT teacher head0.328
Teacher spread0.281 · 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

Citations36
Published2019
Admission routes1
Has abstractyes

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Same venueProceedings of the National Academy of SciencesSame topicOsteomyelitis and Bone Disorders ResearchFrench-language works237,207