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Record W2233837568 · doi:10.1212/nxg.0000000000000009

Loss-of-function mutations in <i>RAB39B</i> are associated with typical early-onset Parkinson disease

2015· article· en· W2233837568 on OpenAlexfundno aff
Suzanne Lesage, José Brás, Florence Cormier‐Dequaire, Christel Condroyer, Aude Nicolas, Lee Darwent, Rita Guerreiro, Elisa Majounie, Monica Federoff, Peter Heutink, Nicholas Wood, Thomas Gasser, John Hardy, François Tison, Andrew Singleton, Alexis Brice

Bibliographic record

VenueNeurology Genetics · 2015
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicCellular transport and secretion
Canadian institutionsnot available
FundersNational Institute on AgingH. Lundbeck A/SMedical Research CouncilFondation Roger de SpoelberchParkinson VerenigingUniversité de BordeauxEberhard Karls Universität TübingenInstitut National de la Santé et de la Recherche MédicaleGlaxoSmithKlineDeutsches Zentrum für Neurodegenerative ErkrankungenCentre Hospitalier Universitaire de BordeauxAssociation France ParkinsonAgence Nationale de la RechercheCentre National de la Recherche ScientifiqueValeant Pharmaceuticals InternationalFONDATION ALZHEIMERMichael J. Fox Foundation for Parkinson's ResearchTeva Pharmaceutical IndustriesNational Institutes of HealthU.S. Department of Health and Human Services
KeywordsMissense mutationLoss functionRabMutationGeneticsBiologyGuanosineGeneIntellectual disabilityGTPasePhenotype

Abstract

fetched live from OpenAlex

Rab proteins are small molecular weight guanosine triphosphatases involved in the regulation of vesicular trafficking.1 Three of 4 X-linked RAB genes are specific to the brain, including RAB39B . Recently, Wilson et al.2 reported that mutations in RAB39B cause X-linked intellectual disability (ID) and pathologically confirmed Parkinson disease (PD). They identified a ∼45-kb deletion resulting in the complete loss of RAB39B in an Australian kindred and a missense mutation in a large Wisconsin kindred. Here, we report an additional affected man with typical PD and mild mental retardation harboring a new truncating mutation in RAB39B . Acknowledgment: The authors are grateful to the patients and their families. They thank Merle Ruberg for critical reading of the manuscript and the DNA and Cell Bank of ICM for sample preparation.

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 score0.004
Threshold uncertainty score0.015

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0000.001
Meta-epidemiology (narrow)0.0020.000
Meta-epidemiology (broad)0.0010.000
Bibliometrics0.0010.001
Science and technology studies0.0010.001
Scholarly communication0.0000.000
Open science0.0010.001
Research integrity0.0010.001
Insufficient payload (model declined to judge)0.0040.002

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 head0.015
GPT teacher head0.222
Teacher spread0.207 · 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

Citations93
Published2015
Admission routes1
Has abstractyes

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