SOX1

Chr 13

SRY-box transcription factor 1

The SOX1 protein is a transcription factor that keeps neural cells undifferentiated during development by suppressing neuronal differentiation and counteracting proneural proteins. Mutations in SOX1 cause microphthalmia with brain and digit anomalies, inherited in an autosomal recessive pattern. This condition affects multiple organ systems including the eyes, brain, and limbs during early development.

Summary from RefSeq, UniProt
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0
Active trials
55
Pubs (1 yr)
118
P/LP submissions
0%
P/LP missense
0.95
LOEUF
LOF
Mechanism· predicted
Clinical SummarySOX1
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.62) — some intolerance to loss-of-function variants.
📋
ClinVar Variants
118 unique Pathogenic / Likely Pathogenic· 82 VUS of 214 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.95LOEUF
pLI 0.623
Z-score 1.63
OE 0.00 (0.000.95)
Moderately constrained

Typical tolerance to LoF variation

Missense Constraint
0.58Z-score
OE missense 0.82 (0.681.00)
71 obs / 86.1 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.00 (0.000.95)
00.351.4
Missense OE0.82 (0.681.00)
00.61.4
Synonymous OE1.40
01.21.6
LoF obs/exp: 0 / 3.1Missense obs/exp: 71 / 86.1Syn Z: -1.96
DN
0.5477th %ile
GOF
0.4185th %ile
LOF
0.81top 5%

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

LOFprediction above median

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

ClinVar Variant Classifications

214 submitted variants in ClinVar

Classification Summary

Pathogenic115
Likely Pathogenic3
VUS82
Likely Benign9
Benign4
115
Pathogenic
3
Likely Pathogenic
82
VUS
9
Likely Benign
4
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
115
0
115
Likely Pathogenic
0
0
3
0
3
VUS
0
73
9
0
82
Likely Benign
0
0
4
5
9
Benign
0
0
1
3
4
Total0731328213

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

View in ClinVar →

Protein Context — Lollipop Plot

SOX1 · 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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