SYNE1

Chr 6ARAD

spectrin repeat containing nuclear envelope protein 1

Also known as: 8B, AMC3, AMCM, ARCA1, C6orf98, CPG2, EDMD4, KASH1

This gene encodes a spectrin repeat protein that localizes to the nuclear outer membrane in skeletal muscle, smooth muscle, and lymphocytes. Mutations cause autosomal recessive spinocerebellar ataxia 8 (cerebellar ataxia type 1), arthrogryposis multiplex congenita type 3, and autosomal dominant Emery-Dreifuss muscular dystrophy type 4. The predicted mechanism involves gain-of-function effects from mutations affecting this nuclear membrane protein.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismAR/ADLOEUF 0.423 OMIM phenotypes
Clinical SummarySYNE1
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Gene-Disease Validity (ClinGen)
arthrogryposis multiplex congenita 3, myogenic type · ARModerate

Moderate evidence — consider for supplementary testing

2 total gene-disease associations curated

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
33 unique Pathogenic / Likely Pathogenic· 313 VUS of 700 total submissions
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GeneReview available — SYNE1
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.42LOEUF
pLI 0.000
Z-score 12.81
OE 0.37 (0.330.42)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
-0.29Z-score
OE missense 1.01 (0.991.04)
4465 obs / 4410.7 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.37 (0.330.42)
00.351.4
Missense OE1.01 (0.991.04)
00.61.4
Synonymous OE1.05
01.21.6
LoF obs/exp: 182 / 487.0Missense obs/exp: 4465 / 4410.7Syn Z: -1.64
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongSYNE1-related spinocerebellar ataxiaLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.73top 25%
GOF
0.81top 10%
LOF
0.1697th %ile

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

Literature Evidence

DNMoreover, the apparent dominance of SYNE mutations suggests a dominant-negative effect, whereby partially compromised nesprins saturate available SUN proteins while failing to bind emerin and/or lamin A/C correctly.PMID:17761684

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

700 submitted variants in ClinVar

Classification Summary

Pathogenic23
Likely Pathogenic10
VUS313
Likely Benign185
Benign47
Conflicting56
23
Pathogenic
10
Likely Pathogenic
313
VUS
185
Likely Benign
47
Benign
56
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
16
0
7
0
23
Likely Pathogenic
9
1
0
0
10
VUS
3
276
21
13
313
Likely Benign
0
14
62
109
185
Benign
0
24
3
20
47
Conflicting
56
Total2831593142634

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

View in ClinVar →

Protein Context — Lollipop Plot

SYNE1 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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Clinical Literature
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