SHANK1

Chr 19

SH3 and multiple ankyrin repeat domains 1

Also known as: SPANK-1, SSTRIP, synamon

This protein functions as a scaffold in the postsynaptic density of excitatory synapses, interconnecting glutamate receptors with the actin cytoskeleton and organizing dendritic spine structure. Deletions cause autism spectrum disorder, particularly in males, with X-linked inheritance. The gene is highly constrained against loss-of-function variation (pLI >0.99), indicating that such variants are likely to be pathogenic.

OMIMResearchSummary from RefSeq, UniProt
LOFmechanismLOEUF 0.18
Clinical SummarySHANK1
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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.
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ClinVar Variants
7 unique Pathogenic / Likely Pathogenic· 166 VUS of 200 total submissions
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.18LOEUF
pLI 1.000
Z-score 7.07
OE 0.10 (0.060.18)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
3.64Z-score
OE missense 0.69 (0.650.73)
738 obs / 1073.8 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.10 (0.060.18)
00.351.4
Missense OE0.69 (0.650.73)
00.61.4
Synonymous OE1.03
01.21.6
LoF obs/exp: 7 / 71.5Missense obs/exp: 738 / 1073.8Syn Z: -0.58
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongSHANK1-related autismLOFAD
DN
0.3793th %ile
GOF
0.5366th %ile
LOF
0.81top 5%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median · 100% of P/LP variants are LoF · LOEUF 0.18

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

200 submitted variants in ClinVar

Classification Summary

Pathogenic3
Likely Pathogenic4
VUS166
Likely Benign26
3
Pathogenic
4
Likely Pathogenic
166
VUS
26
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
0
0
0
3
Likely Pathogenic
4
0
0
0
4
VUS
0
165
1
0
166
Likely Benign
0
21
2
3
26
Benign
0
0
0
0
0
Total718633199

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

View in ClinVar →

Protein Context — Lollipop Plot

SHANK1 · 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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