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Record W4405220612 · doi:10.1101/2024.12.10.24318787

MDSGene update and expansion: Clinical and genetic spectrum of <i>LRRK2</i> variants in Parkinson’s disease

2024· preprint· en· W4405220612 on OpenAlexaff
Shen‐Yang Lim, Alissa Buhrmann, Fenja L. Fahrig, Carolin Gabbert, Natascha Bahr, Harutyun Madoev, Connie Marras, Christine Klein, Katja Lohmann

Bibliographic record

VenuemedRxiv · 2024
Typepreprint
Languageen
FieldMedicine
TopicParkinson's Disease Mechanisms and Treatments
Canadian institutionsUniversity of TorontoUniversity Health Network
Fundersnot available
KeywordsLRRK2DiseaseBiologyGeneticsParkinson's diseasePathogenicityPhenotypeGenetic testingGenotypeGeneMedicineInternal medicine

Abstract

fetched live from OpenAlex

ABSTRACT Pathogenic variants in the LRRK2 gene are one of the most commonly identifiable monogenic causes of Parkinsońs disease (PD, PARK-LRRK2). This systematic MDSGene literature review comprehensively summarizes published demographic, clinical, and genetic findings related to potentially pathogenic LRRK2 variants ( https://www.mdsgene.org/ ). Recent insights on LRRK2’s kinase activity have been incorporated for pathogenicity scoring. Data on 7,885 individuals with 292 different variants were curated, including 3,296 patients with PD carrying 205 different potentially disease-causing LRRK2 variants. The initial MDSGene review covered only 724 patients carrying 23 different LRRK2 variants. Missingness of phenotypic data in the literature was high, hampering the identification of detailed genotype-phenotype correlations. Notably, the median age at onset in the patients with available information was 56 years, with approximately one-third having PD onset <50 years. Tremor was the most frequently reported initial symptom and more frequent than reported in other dominantly inherited forms of PD. Of the 205 potentially disease-causing variants, 14 (6.8%) were classified as pathogenic, 8 (3.9%) as likely pathogenic, and the remaining 183 (89.3%) as variants of uncertain significance (VUS). The pathogenic p.G2019S variant was the most frequent pathogenic variant, followed by p.R1441G and p.R1441C, accounting for >80% of patients, with Tunisia, Spain, and Italy contributing about half of patients. This systematic review represents the largest database on PARK-LRRK2 to date and provides an important resource to improve precision medicine. Given their high frequency, a better interpretation of the pathogenicity of VUS is needed for selection and stratification of patients in clinical trials.

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)0.003
metaresearch head score (Gemma)0.010
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: none
Teacher disagreement score0.016
Threshold uncertainty score0.020

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0030.010
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0020.002
Bibliometrics0.0160.007
Science and technology studies0.0000.000
Scholarly communication0.0020.002
Open science0.0010.002
Research integrity0.0010.001
Insufficient payload (model declined to judge)0.0060.003

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 head0.026
GPT teacher head0.303
Teacher spread0.277 · 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

Citations1
Published2024
Admission routes1
Has abstractyes

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