The SYNPO2 protein binds and bundles actin filaments and serves as an adapter protein at sarcomeric Z lines, linking myofibers to the sarcolemma and contributing to muscle fiber assembly and maintenance. Mutations cause autosomal recessive myopathy with characteristic features including muscle weakness and structural abnormalities of muscle fibers. The gene is highly constrained against loss-of-function variants, indicating intolerance to complete protein loss.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
9
Pubs (1 yr)
21
P/LP submissions
0%
P/LP missense
0.63
LOEUF
Multiple*
Mechanism· predicted
Clinical SummarySYNPO2
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
21 unique Pathogenic / Likely Pathogenic· 182 VUS of 223 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.63LOEUF
pLI 0.000
Z-score 3.57
OE 0.43 (0.300.63)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.04Z-score
OE missense 1.00 (0.941.06)
688 obs / 690.9 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.43 (0.300.63)
00.351.4
Missense OE1.00 (0.941.06)
00.61.4
Synonymous OE0.98
01.21.6
LoF obs/exp: 20 / 46.2Missense obs/exp: 688 / 690.9Syn Z: 0.31
DN
0.7034th %ile
GOF
0.6639th %ile
LOF
0.3745th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative and gain-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

DNprediction above median
GOFprediction above median

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.

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

ClinVar Variant Classifications

223 submitted variants in ClinVar

Classification Summary

Pathogenic20
Likely Pathogenic1
VUS182
Likely Benign5
Benign6
20
Pathogenic
1
Likely Pathogenic
182
VUS
5
Likely Benign
6
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
20
0
20
Likely Pathogenic
0
0
1
0
1
VUS
1
178
3
0
182
Likely Benign
0
3
1
1
5
Benign
0
3
0
3
6
Total1184254214

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

View in ClinVar →

Protein Context — Lollipop Plot

SYNPO2 · protein map & ClinVar variants

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

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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