SPOP

Chr 17AD

speckle type BTB/POZ protein

Also known as: BTBD32, NEDMACE, NEDMIDF, NSDVS1, NSDVS2, TEF2

The SPOP protein functions as a component of a cullin-RING-based E3 ubiquitin ligase complex that targets specific proteins for degradation or regulation, playing essential roles in transcriptional regulation, DNA replication, and cell proliferation. Mutations cause Nabais Sa-de Vries syndrome types 1 and 2 with autosomal dominant inheritance. This gene is highly constrained against loss-of-function variation in the population, indicating that such mutations are likely to have significant clinical consequences.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismADLOEUF 0.142 OMIM phenotypes
Clinical SummarySPOP
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Gene-Disease Validity (ClinGen)
neurodevelopmental disorder with microcephaly and dysmorphic facies · ADModerate

Moderate evidence — consider for supplementary testing

2 total gene-disease associations curated

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.
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Clinical Trials
3 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.14LOEUF
pLI 0.999
Z-score 4.21
OE 0.00 (0.000.14)
Highly constrained

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

Missense Constraint
4.14Z-score
OE missense 0.21 (0.170.27)
46 obs / 218.1 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.00 (0.000.14)
00.351.4
Missense OE0.21 (0.170.27)
00.61.4
Synonymous OE1.11
01.21.6
LoF obs/exp: 0 / 20.7Missense obs/exp: 46 / 218.1Syn Z: -0.77
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongSPOP-related neurodevelopmental disorder, gain of functionGOFAD
strongSPOP-related neurodevelopmental disorder, dominant negativeDNAD
DN
0.3494th %ile
GOF
0.3690th %ile
LOF
0.75top 10%

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

LOFprediction above median · LOEUF 0.14
GOF1 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

DNRecurrent somatic variants in SPOP are cancer specific; endometrial and prostate cancers result from gain-of-function and dominant-negative effects toward BET proteins, respectively.PMID:32109420
GOFRecurrent somatic variants in SPOP are cancer specific; endometrial and prostate cancers result from gain-of-function and dominant-negative effects toward BET proteins, respectively.PMID:32109420

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

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

SPOP · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
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