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Record W2775797405 · doi:10.1212/nxg.0000000000000200

<i>CDKL5</i> variants

2017· article· he· W2775797405 on OpenAlex

Why this work is in the frame

A frame that forgets how it found something cannot be audited. These are the routes that admitted this work.

fundA Canadian funder is recorded on the work.
no affNo Canadian affiliation: this work is invisible to an affiliation-only frame.
No Canadian affiliation. An affiliation-only frame, the usual design, would never have seen this work. It is one of the works that make the case for inverting the frame.

Bibliographic record

VenueNeurology Genetics · 2017
Typearticle
Languagehe
FieldBiochemistry, Genetics and Molecular Biology
TopicGenetics and Neurodevelopmental Disorders
Canadian institutionsnot available
FundersNational Institutes of HealthUniversity of Colorado School of Medicine, Anschutz Medical CampusInternational Foundation for CDKL5 ResearchMax-Planck-GesellschaftBundesministerium für Bildung und ForschungEunice Kennedy Shriver National Institute of Child Health and Human DevelopmentEconomic and Social Research CouncilCurtin University of TechnologyRosetrees TrustChildren's Hospital ColoradoMallinckrodt PharmaceuticalsActelion PharmaceuticalsNational Health and Medical Research CouncilLouLou FoundationRett Syndrome Association of AustraliaEdimer PharmaceuticalsQuestcor PharmaceuticalsCitizens United for Research in EpilepsyUniversity of PennsylvaniaRett Syndrome Research TrustAveXisBiogen
KeywordsMissense mutationBiologyNonsenseExonGeneticsRNA splicingPopulationAlternative splicingAlleleGeneComputational biologyBioinformaticsMutationMedicine

Abstract

fetched live from OpenAlex

<h3>Objective:</h3> To provide new insights into the interpretation of genetic variants in a rare neurologic disorder, CDKL5 deficiency, in the contexts of population sequencing data and an updated characterization of the <i>CDKL5</i> gene. <h3>Methods:</h3> We analyzed all known potentially pathogenic <i>CDKL5</i> variants by combining data from large-scale population sequencing studies with <i>CDKL5</i> variants from new and all available clinical cohorts and combined this with computational methods to predict pathogenicity. <h3>Results:</h3> The study has identified several variants that can be reclassified as benign or likely benign. With the addition of novel <i>CDKL5</i> variants, we confirm that pathogenic missense variants cluster in the catalytic domain of CDKL5 and reclassify a purported missense variant as having a splicing consequence. We provide further evidence that missense variants in the final 3 exons are likely to be benign and not important to disease pathology. We also describe benign splicing and nonsense variants within these exons, suggesting that isoform hCDKL5_5 is likely to have little or no neurologic significance. We also use the available data to make a preliminary estimate of minimum incidence of CDKL5 deficiency. <h3>Conclusions:</h3> These findings have implications for genetic diagnosis, providing evidence for the reclassification of specific variants previously thought to result in CDKL5 deficiency. Together, these analyses support the view that the predominant brain isoform in humans (hCDKL5_1) is crucial for normal neurodevelopment and that the catalytic domain is the primary functional domain.

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.

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)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Bench or experimental · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.740
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0010.001
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0010.001
Scholarly communication0.0000.000
Open science0.0020.001
Research integrity0.0010.000
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 head0.014
GPT teacher head0.251
Teacher spread0.237 · 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