UNC13A

Chr 19ADAR

unc-13 homolog A

Also known as: IDDSF, Munc13-1, NEDHES, NEDSMS

The protein binds to phorbol esters and diacylglycerol and regulates neurotransmitter release at synapses. Loss-of-function mutations cause neurodevelopmental disorders characterized by intellectual disability, seizures, hypotonia, absent or delayed speech, movement abnormalities, and dysmorphic features, with both autosomal dominant and autosomal recessive inheritance patterns reported. The gene is extremely intolerant to loss-of-function variation, consistent with its critical role in synaptic function.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
MultiplemechanismAD/ARLOEUF 0.163 OMIM phenotypes
Clinical SummaryUNC13A
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
20 unique Pathogenic / Likely Pathogenic· 255 VUS of 437 total submissions
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GeneReview available — UNC13A
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.16LOEUF
pLI 1.000
Z-score 8.39
OE 0.09 (0.050.16)
Highly constrained

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

Missense Constraint
5.63Z-score
OE missense 0.50 (0.470.54)
513 obs / 1018.3 exp
Constrained

Extremely missense-constrained (top ~0.01%)

Observed / Expected Ratios
LoF OE0.09 (0.050.16)
00.351.4
Missense OE0.50 (0.470.54)
00.61.4
Synonymous OE1.00
01.21.6
LoF obs/exp: 9 / 99.1Missense obs/exp: 513 / 1018.3Syn Z: -0.03
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
moderateUNC13A-related congenital epileptic encephalopathy and severe neuromuscular disorderLOFAR
moderateUNC13A-related neurodevelopmental disorder with ataxia and tremor or dyskinetic movementsGOFAD
DN
0.4388th %ile
GOF
0.6248th %ile
LOF
0.66top 25%

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

LOFprediction above median · LOEUF 0.16

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. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

437 submitted variants in ClinVar

Classification Summary

Pathogenic17
Likely Pathogenic3
VUS255
Likely Benign115
Benign28
Conflicting5
17
Pathogenic
3
Likely Pathogenic
255
VUS
115
Likely Benign
28
Benign
5
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
1
5
11
0
17
Likely Pathogenic
0
2
1
0
3
VUS
24
220
11
0
255
Likely Benign
0
8
18
89
115
Benign
0
4
6
18
28
Conflicting
5
Total2523947107423

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

View in ClinVar →

Protein Context — Lollipop Plot

UNC13A · 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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