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Record W3165718938 · doi:10.1038/s41431-021-00900-2

A MT-TL1 variant identified by whole exome sequencing in an individual with intellectual disability, epilepsy, and spastic tetraparesis

2021· article· en· W3165718938 on OpenAlexfundno aff
Elke de Boer, Charlotte W. Ockeloen, Leslie Matalonga, Rita Horváth, Enzo Cohen, Isabel Cuesta, Daniel Danis, Anne‐Sophie Denommé‐Pichon, Yannis Duffourd, Christian Gilissen, Mridul Johari, Steven Laurie, Shuang Li, Isabelle Nelson, Sophia Peters, Prasanth Sivakumar, Peter Robinson, Karolis Šablauskas, Marco Savarese, Wouter Steyaert, Ana Töpf, Joeri K. van der Velde, Antonio Vitobello, Richard J. Rodenburg, Marieke J. H. Coenen, Mirian C. H. Janssen, Dylan Henssen, Christian Gilissen, Siddharth Banka, Elisa Benetti, Giorgio Casari, Andrea Ciolfi, Jill Clayton‐Smith, Bruno Dallapiccola, Kornelia Ellwanger, Laurence Faivre, Holm Graeßner, Tobias B. Haack, Anna Hammarsjö, Markéta Havlovicová, Alexander Hoischen, Anne Hugon, Adam Jackson, Anna Lindstrand, Estrella López‐Martín, Milan Macek, Manuela Morleo, Vicenzo Nigro, Ann Nordgren, Maria Pettersson, Michele Pinelli, Simone Pizzi, Manuel Posada de la Paz, Francesca Clementina Radio, Alessandra Renieri, Caroline Rooryck, Lukáš Ryba, Martin Schwarz, Marco Tartaglia, Christel Thauvin, Annalaura Torella, Alain Verloès, Lisenka E.L.M. Vissers, Pavel Votýpka, Klea Vyshka, Birte Zurek, Aurélien Trimouille

Bibliographic record

VenueEuropean Journal of Human Genetics · 2021
Typearticle
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicGenomics and Rare Diseases
Canadian institutionsnot available
FundersInstituto de Salud Carlos IIIInstitute of GeneticsDirectorate for Biological SciencesUniversité de ParisAssistance publique-Hôpitaux de ParisRadboud Universitair Medisch CentrumLily FoundationEvelyn TrustUniverzita Karlova v PrazeEberhard Karls Universität TübingenEuropean CommissionAtaxia UKThird Health ProgrammeNewton FundUniversità degli Studi di SienaRadboud UniversiteitMedical Research CouncilOspedale Pediatrico Bambino GesùCentre Hospitalier Universitaire de BordeauxNederlandse Organisatie voor Wetenschappelijk OnderzoekWellcome TrustKarolinska InstitutetInstitut National de la Santé et de la Recherche Médicale
KeywordsHeteroplasmyExome sequencingProbandMitochondrial DNAEpilepsyGeneticsIntellectual disabilityTetraparesisMedicineBiologyPhenotypeMutationPsychiatry

Abstract

fetched live from OpenAlex

The genetic etiology of intellectual disability remains elusive in almost half of all affected individuals. Within the Solve-RD consortium, systematic re-analysis of whole exome sequencing (WES) data from unresolved cases with (syndromic) intellectual disability (n = 1,472 probands) was performed. This re-analysis included variant calling of mitochondrial DNA (mtDNA) variants, although mtDNA is not specifically targeted in WES. We identified a functionally relevant mtDNA variant in MT-TL1 (NC_012920.1:m.3291T > C; NC_012920.1:n.62T > C), at a heteroplasmy level of 22% in whole blood, in a 23-year-old male with severe intellectual disability, epilepsy, episodic headaches with emesis, spastic tetraparesis, brain abnormalities, and feeding difficulties. Targeted validation in blood and urine supported pathogenicity, with heteroplasmy levels of 23% and 58% in index, and 4% and 17% in mother, respectively. Interestingly, not all phenotypic features observed in the index have been previously linked to this MT-TL1 variant, suggesting either broadening of the m.3291T > C-associated phenotype, or presence of a co-occurring disorder. Hence, our case highlights the importance of underappreciated mtDNA variants identifiable from WES data, especially for cases with atypical mitochondrial phenotypes and their relatives in the maternal line.

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)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
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 score0.787
Threshold uncertainty score0.619

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
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 head0.021
GPT teacher head0.244
Teacher spread0.223 · 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.

The models applied no category: nothing in the taxonomy fit this work.
Study designBench or experimental
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

Citations11
Published2021
Admission routes1
Has abstractyes

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