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Record W4409119522 · doi:10.1002/ajmg.a.64080

Tall Stature and Scoliosis Associated With a Novel Homozygous Loss‐of‐Function Missense Variant in <i>NPR3</i>

2025· article· en· W4409119522 on OpenAlexafffund
Pierre Moffatt, Chantal Janelle, Valancy Miranda, Ghalib Bardai, Frank Rauch

Bibliographic record

VenueAmerican Journal of Medical Genetics Part A · 2025
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGenetics and Neurodevelopmental Disorders
Canadian institutionsMcGill UniversityShriners Hospitals for Children - Canada
FundersFondation Mirella et Lino Saputo
KeywordsShort statureScoliosisMissense mutationBiologyInternal medicinePhenotypeMedicineEndocrinologyGeneticsGene

Abstract

fetched live from OpenAlex

NPR3-related tall stature is characterized by tall stature, elongated big toes, and additional epiphyses in hand and foot bones. The condition is caused by biallelic loss-of-function variants affecting natriuretic peptide receptor 3 (NPR3). Five individuals from four different families have been reported. Here we describe three siblings with NPR3-related tall stature who were tall (height z-scores between +2.9 and + 4.9) and had markedly elongated proximal and middle phalanges. Two siblings had additional epiphyses in phalangeal and metacarpal bones. All three siblings developed scoliosis, requiring spinal fusion surgery in one individual. Lumbar spine bone mineral density appeared low considering the tall stature. Sequencing of a skeletal disorders gene panel in one sibling revealed a homozygous missense variant in NPR3 (NM_001204375.2; c.382C>T; p.Pro128Ser). Sanger sequencing demonstrated the same homozygous variant in the other siblings. In vitro functional testing in MC3T3-E1 preosteoblastic cells showed that NPR3 carrying the p.Pro128Ser variant was expressed but was retained in the endoplasmic reticulum, leading to loss of NPR3 function. In conclusion, the novel homozygous p.Pro128Ser loss-of-function variant in NPR3 led to the typical features of NPR3-related tall stature and, in addition, was associated with scoliosis. These observations expand the genotypic and phenotypic spectrum of NPR3-related tall stature.

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: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.003
Threshold uncertainty score0.006

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0000.001
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0000.001
Bibliometrics0.0010.001
Science and technology studies0.0010.001
Scholarly communication0.0000.000
Open science0.0010.001
Research integrity0.0020.001
Insufficient payload (model declined to judge)0.0020.000

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 head0.004
GPT teacher head0.227
Teacher spread0.223 · 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

Citations0
Published2025
Admission routes2
Has abstractyes

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