MétaCan
Menu
Back to cohort
Record W3009176330 · doi:10.1101/2020.03.06.20030734

Variants in the Niemann-Pick type C gene <i>NPC1</i> are not associated with Parkinson’s disease

2020· preprint· en· W3009176330 on OpenAlexafffund
Bouchra Ouled Amar Bencheikh, Konstantin Senkevich, Uladzislau Rudakou, Eric Yu, Kheireddin Mufti, Jennifer A. Ruskey, Farnaz Asayesh, Sandra B. Laurent, Dan Spiegelman, Stanley Fahn, Cheryl Waters, Oury Monchi, Yves Dauvilliers, Alberto J. Espay, Nicolas Dupré, Lior Greenbaum, Sharon Hassin‐Baer, Guy A. Rouleau, Roy N. Alcalay, Edward A. Fon, Ziv Gan‐Or

Bibliographic record

VenuemedRxiv · 2020
Typepreprint
Languageen
FieldMedicine
TopicLysosomal Storage Disorders Research
Canadian institutionsUniversité LavalOntario Brain InstituteUniversity of CalgaryMcGill UniversityCentre Hospitalier de l’Université de MontréalMontreal Neurological Institute and Hospital
FundersNational Institutes of HealthCanada First Research Excellence FundCanadian Institutes of Health ResearchParkinson CanadaConsortium canadien en neurodégénérescence associée au vieillissementMcGill UniversityParkinson's FoundationBrookdale FoundationFonds de Recherche du Québec - SantéMichael J. Fox Foundation for Parkinson's Research
KeywordsNPC1Niemann–Pick diseaseParkinson's diseaseNiemann–Pick disease, type CLRRK2BiologyDiseaseCompound heterozygosityLysosomal storage diseaseGlucocerebrosidaseGeneticsMedicineGeneMutationPathology

Abstract

fetched live from OpenAlex

Abstract Biallelic variants in NPC1 , a lysosomal gene coding for a transmembrane protein involved in cholesterol trafficking, may cause Niemann-Pick disease type C (NPC). A few cases of NPC1 mutation carriers have been reported with a Parkinson’s disease (PD) presentation. In addition, pathological studies demonstrated phosphorylated alpha-synuclein and Lewy pathology in brains of NPC patients. Therefore, we aimed to examine whether NPC1 genetic variants may be associated with PD. Full sequencing of NPC1 was performed in 2,657 PD patients and 3,647 controls from three cohorts, using targeted sequencing with molecular inversion probes. A total of 9 common variants and 126 rare variants were identified across the three cohorts. To examine association with PD, regression models adjusted for age, sex and origin were performed for common variants, and optimal sequence Kernel association test (SKAT-O) was performed for rare variants. After correction for multiple comparisons, common and rare NPC1 variants were not associated with PD. Our results do not support a link between heterozygous variants in NPC1 and PD.

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)0.001
metaresearch head score (Gemma)0.002
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: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.005
Threshold uncertainty score0.016

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0010.002
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.000
Bibliometrics0.0010.001
Science and technology studies0.0010.000
Scholarly communication0.0010.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0050.001

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 head0.056
GPT teacher head0.300
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 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
Published2020
Admission routes2
Has abstractyes

Explore more

Same venuemedRxivSame topicLysosomal Storage Disorders ResearchFrench-language works237,207