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Record W3157437255 · doi:10.1111/epi.16913

Homozygous <i>SCN1B</i> variants causing early infantile epileptic encephalopathy 52 affect voltage‐gated sodium channel function

2021· article· en· W3157437255 on OpenAlexafffund
Marcello Scala, Stéphanie Efthymiou, Tipu Sultan, Jolien De Waele, Marta Panciroli, Vincenzo Salpietro, Reza Maroofian, Pasquale Striano, Filip Van Petegem, Henry Houlden, Frank Bosmans

Bibliographic record

VenueEpilepsia · 2021
Typearticle
Languageen
FieldMedicine
TopicCardiac electrophysiology and arrhythmias
Canadian institutionsUniversity of British Columbia
FundersNational Institute of Neurological Disorders and StrokeMedical Research CouncilMuscular Dystrophy UKRosetrees TrustCanadian Institutes of Health ResearchNational Institute for Health and Care ResearchAtaxia UKBrain Research UKMultiple System Atrophy TrustMuscular Dystrophy Association
KeywordsSodium channelAffect (linguistics)EpilepsyEncephalopathyMedicineSodiumInternal medicineChemistryPsychologyPsychiatryCommunication

Abstract

fetched live from OpenAlex

Abstract We identified nine patients from four unrelated families harboring three biallelic variants in SCN1B (NM_001037.5: c.136C&gt;T; p.[Arg46Cys], c.178C&gt;T; p.[Arg60Cys], and c.472G&gt;A; p.[Val158Met]). All subjects presented with early infantile epileptic encephalopathy 52 (EIEE52), a rare, severe developmental and epileptic encephalopathy featuring infantile onset refractory seizures followed by developmental stagnation or regression. Because SCN1B influences neuronal excitability through modulation of voltage‐gated sodium (Na V ) channel function, we examined the effects of human SCN1B R46C ( β1 R46C ), SCN1B R60C ( β1 R60C ), and SCN1B V158M ( β1 V158M ) on the three predominant brain Na V channel subtypes Na V 1.1 ( SCN1A ), Na V 1.2 ( SCN2A ), and Na V 1.6 ( SCN8A ). We observed a shift toward more depolarizing potentials of conductance–voltage relationships (Na V 1.2/ β1 R46C , Na V 1.2/ β1 R60C , Na V 1.6/ β1 R46C , Na V 1.6/ β1 R60C , and Na V 1.6/ β1 V158M ) and channel availability (Na V 1.1/ β1 R46C , Na V 1.1/ β1 V158M , Na V 1.2/ β1 R46C , Na V 1.2/ β1 R60C , and Na V 1.6/ β1 V158M ), and detected a slower recovery from fast inactivation for Na V 1.1/ β1 V158M . Combined with modeling data indicating perturbation‐induced structural changes in β1 , these results suggest that the SCN1B variants reported here can disrupt normal Na V channel function in the brain, which may contribute to EIEE52.

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 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 score0.862
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.000
Bibliometrics0.0000.001
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.001
Insufficient payload (model declined to judge)0.0010.001

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 head0.009
GPT teacher head0.237
Teacher spread0.228 · 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.

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

Citations23
Published2021
Admission routes2
Has abstractyes

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