SCN2A

Chr 2AD

sodium voltage-gated channel alpha subunit 2

Also known as: BFIC3, BFIS3, BFNIS, DEE11, EA9, EIEE11, HBA, HBSCI

The protein encoded by this gene forms voltage-gated sodium channels that generate and propagate action potentials in neurons. Mutations cause developmental and epileptic encephalopathy 11, benign familial infantile seizures type 3, and episodic ataxia type 9 through an autosomal dominant inheritance pattern. Disease mechanisms are variant-dependent, with early infantile seizures typically caused by gain-of-function mutations and later-onset seizures often resulting from loss-of-function mutations.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
GOF/LOFmechanismADLOEUF 0.133 OMIM phenotypes
VCEP Guidelines: Epilepsy Sodium ChannelReleased
View SpecificationsClinGen Panel
Clinical SummarySCN2A
🧬
Gene-Disease Validity (ClinGen)
complex neurodevelopmental disorder · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
📋
ClinVar Variants
132 unique Pathogenic / Likely Pathogenic· 280 VUS of 581 total submissions
💊
Clinical Trials
3 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — SCN2A
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.13LOEUF
pLI 1.000
Z-score 7.92
OE 0.06 (0.030.13)
Highly constrained

Among the most LoF-intolerant genes (~top 3%)

Missense Constraint
6.46Z-score
OE missense 0.44 (0.410.48)
469 obs / 1061.0 exp
Constrained

Extremely missense-constrained (top ~0.01%)

Observed / Expected Ratios
LoF OE0.06 (0.030.13)
00.351.4
Missense OE0.44 (0.410.48)
00.61.4
Synonymous OE1.01
01.21.6
LoF obs/exp: 5 / 82.7Missense obs/exp: 469 / 1061.0Syn Z: -0.13
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveSCN2A-related nonspecific severe intellectual disabilityLOFAD
definitiveSCN2A-related infantile epileptic encephalopathyOTHERAD
Mechanism Note (variant-dependent)
GOFLOF— mechanism depends on specific variant

Nav1.2 is monomeric. Early-onset DEE variants (<3 months) are GOF with increased persistent current. Later-onset epilepsy and ASD/ID variants are LOF. Mechanism is age-of-onset and variant-dependent.

DN
0.5772th %ile
GOF
0.80top 10%
LOF
0.51top 25%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function, gain-of-function and dominant-negative). The Badonyi & Marsh model scores gain-of-function highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports loss-of-function (haploinsufficiency). Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOF1 literature citation · 34% of P/LP variants are LoF · LOEUF 0.13
GOFprediction above median · 1 literature citation
DN1 literature citation

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

DNElectrophysiologic studies in HEK293 cells showed that the R102X mutant protein was nonfunctional when expressed in isolation, and shifted the voltage dependence of inactivation of wildtype channels in the hyperpolarizing direction, consistent with a dominant-negative effect.PMID:15028761
GOFThe third new proband carries the same de novo SCN2A gain-of-function mutation as our first published case (p.Ala263Val).PMID:26645390
LOFWe discuss the effects of haploinsufficiency of SCN2A and SCN3A on the genetic basis of neurodevelopmental and neurobehavioral disorders and we propose that this haploinsufficiency may be associated not only with epilepsy, but also with autistic features.PMID:24080482

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

581 submitted variants in ClinVar

Classification Summary

Pathogenic69
Likely Pathogenic63
VUS280
Likely Benign129
Benign17
Conflicting17
69
Pathogenic
63
Likely Pathogenic
280
VUS
129
Likely Benign
17
Benign
17
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
30
21
18
0
69
Likely Pathogenic
15
44
4
0
63
VUS
4
259
12
5
280
Likely Benign
0
2
44
83
129
Benign
0
2
7
8
17
Conflicting
17
Total493288596575

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

SCN2A · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Neurodevelopmental disorders-the history and future of a diagnostic concept
.
Morris-Rosendahl DJ et al.·Dialogues Clin Neurosci
2020Review
Progress in Understanding and Treating SCN2A-Mediated Disorders.
Sanders SJ et al.·Trends Neurosci
2018Review
The phenotypic spectrum of SCN2A-related epilepsy.
Reynolds C et al.·Eur J Paediatr Neurol
2020Review
SCN1A-related phenotypes: Epilepsy and beyond.
Scheffer IE et al.·Epilepsia
2019Review
Top 5 results · since 2015Search PubMed ↗