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Record W4406804310 · doi:10.1002/mds.30101

Disco‐Interacting Protein 2 Homolog B <scp>CGG</scp> Repeat Expansion in Siblings with Neurodevelopmental Disability and Progressive Movement Disorder

2025· article· en· W4406804310 on OpenAlexafffund
Emilie T. Théberge, Kate Durbano, Diane Demailly, Sophie Huby, Aleksandra Mitina, Yue Yin, Arezoo Mohajeri, Clara D.M. van Karnebeek, Gabriella Horváth, Ryan K. C. Yuen, Karen Usdin, Anna Lehman, Laura Cif, Phillip A. Richmond

Bibliographic record

VenueMovement Disorders · 2025
Typearticle
Languageen
FieldNeuroscience
TopicGenetic Neurodegenerative Diseases
Canadian institutionsSickKids FoundationUniversity of TorontoBC Children's HospitalHospital for Sick ChildrenUniversity of British Columbia
FundersHospital for Sick ChildrenCanadian Institutes of Health ResearchAzrieli FoundationUniversity of TorontoDepartment of Health and Social CareCancer Research UKGenome British ColumbiaCanada's Michael Smith Genome Sciences CentreNational Institute for Health and Care ResearchGenome CanadaMedical Research CouncilChildren’s Hospital of Wisconsin Research InstituteWellcome TrustFondation Brain Canada
KeywordsTrinucleotide repeat expansionPhenotypeGeneticsChoreaDystoniaMovement disordersAtaxiaBiologyMedicineNeuroscienceGeneAlleleDiseaseInternal medicine

Abstract

fetched live from OpenAlex

BACKGROUND: Trinucleotide repeat expansions are an emerging class of genetic variants associated with various movement disorders. Unbiased genome-wide analyses can reveal novel genotype-phenotype associations and provide a diagnosis for patients and families. OBJECTIVE: The aim was to identify the genetic cause of a severe progressive movement disorder phenotype in 2 affected brothers. METHODS: A family of 2 affected brothers and unaffected parents had extensive phenotyping since birth. Whole-genome and long-read sequencing methods characterized genetic variants and methylation status. RESULTS: Two male siblings with a CGG repeat expansion in the 5'-untranslated region (UTR) of disco-interacting protein 2 homolog B (DIP2B) presented with a novel DIP2B phenotype, including neurodevelopmental disability, dysmorphic traits, and a severe progressive movement disorder (chorea, dystonia, and ataxia). CONCLUSIONS: This is the first report of a severe progressive movement disorder phenotype associated with a CGG repeat expansion in the DIP2B 5'-UTR. © 2025 International Parkinson and Movement Disorder Society. This article has been contributed to by U.S. Government employees and their work is in the public domain in the USA.

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)0.000
metaresearch head score (Gemma)0.001
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.265
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.001
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.001
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 head0.009
GPT teacher head0.254
Teacher spread0.244 · 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.

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

Citations5
Published2025
Admission routes2
Has abstractyes

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