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Record W7161997847 · doi:10.82308/18870

The strongman trait: clinical and molecular characterization of a dominant herculean myalgic disorder

2019· dissertation· en· W7161997847 on OpenAlexaboutno aff
Talita Conte

Bibliographic record

Venuenot available
Typedissertation
Languageen
FieldBiochemistry, Genetics and Molecular Biology
TopicBiotin and Related Studies
Canadian institutionsnot available
Fundersnot available
KeywordsEndoplasmic reticulumAlleleGeneMutationTraitWeaknessPhenotype

Abstract

fetched live from OpenAlex

Herculean strength has always fascinated humans. Several studies have suggested that muscular strength is a trait proposing that genetic factors contribute to the development of an increased strength phenotype. Most male French-Canadian settlers that came to Quebec during the French Regime (1608-1760) were soldiers chosen by merchants for their strength. This may have influenced the relative increase in carrier frequencies of certain alleles related to muscle strength that have been passed down through generations in Quebec. We recruited several French-Canadian families with individuals presenting a muscle disorder that we have called the Strongman trait (ST). Affected individuals present with a familial and personal history of superior strength, which is accompanied by myalgia, prolonged contractions and weakness after repetitive contractions as negative symptoms, and superior muscle strength and mass as positive findings. We identified an autosomal dominant mutation segregating in two families in an uncharacterized gene called DC-STAMP Domain Containing 2 (DCST2). By further exploring the function of DCST2, we found that it is localized to the sarcoplasmic reticulum (SR) and forms puncta that lie juxtaposed to the transverse-tubules (TT) in the I-band. We observed that DCST2 formed larger puncta in the muscle of a ST patient and it often colocalized with stromal interaction molecule 1 (STIM1) and ATPase sarcoplasmic/endoplasmic reticulum Ca2+ transporting 1 (SERCA1), a finding that was rarely observed in control muscles. We performed an unbiased approach through a proximity biotinylation (BioID) assay to identify some DCST2’s proximity interactors. Several proteins of the endoplasmic reticulum (ER) were identified as well as important proteins involved with cellular calcium handling. Two of the proteins involved with calcium handling with the highest scores in the BioID were STIM1 and coiled-coil domain containing 47 (CCDC47 or calumin). Considering that these two proteins have been described to have important roles as calcium sensors influencing ER calcium replenishing through the store-operated calcium entry (SOCE), we then performed cellular calcium assays to identify if the mutation in DCST2 could have an effect on cellular calcium transients. We observed that ST differentiating myoblasts elicited lower SR calcium release and we confirmed that this effect is probably due to a defective SERCA activity that is unable to properly reuptake calcium ions from the cytosol back to the SR. We also detected lower extracellular calcium entry by measuring calcium-release calcium activated (CRAC) channels activity. This result indicated that STIM1 is partially dysfunctional to allow proper extracellular calcium entry through the CRAC channels. We also detected that ST myoblasts presented a greater number of larger DCST2 puncta and that this number tended to increase when cells were exposed to calcium deficit stress. These larger DCST2 puncta also contained STIM1 and SERCA1 suggesting that DCST2 is probably involved in the dynamics of STIM1-SERCA1 multi-protein complex formation. DCST2-L759P found in the ST cells appears to lead to an improper complex formation impacting cellular calcium transients. We showed that ST patients that do not carry DCST2 mutations presented overlapping clinical and pathological (larger DCST2 puncta) features as ST patients carrying a DCST2 mutation, underlining that they could be used to support the ST diagnosis in a clinical setting. This thesis presents the original description of the ST phenotype and the identification of its first causal gene while concluding that calcium handling dysfunctions are responsible for this new Herculean myopathy

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: Bench or experimental
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.415
Threshold uncertainty score0.464

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 integrity0.0010.000
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 head0.006
GPT teacher head0.274
Teacher spread0.268 · 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

Citations0
Published2019
Admission routes1
Has abstractyes

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