MICU3

Chr 8

mitochondrial calcium uptake 3

Also known as: EFHA2, hMICU3

MICU3 encodes a tissue-specific calcium sensor that regulates the mitochondrial calcium uniporter channel in the central nervous system and skeletal muscle, forming a heterodimer with MICU1 to control mitochondrial calcium uptake based on cellular calcium levels. Mutations cause autosomal recessive developmental and epileptic encephalopathy with movement abnormalities, typically presenting in infancy or early childhood. The gene is not highly constrained against loss-of-function variants, suggesting that complete loss of protein function may be tolerated in some contexts.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
6
Pubs (1 yr)
80
P/LP submissions
0%
P/LP missense
1.02
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryMICU3
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
80 unique Pathogenic / Likely Pathogenic· 113 VUS of 215 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.02LOEUF
pLI 0.000
Z-score 1.49
OE 0.68 (0.461.02)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
0.22Z-score
OE missense 0.96 (0.861.07)
228 obs / 237.5 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.68 (0.461.02)
00.351.4
Missense OE0.96 (0.861.07)
00.61.4
Synonymous OE1.26
01.21.6
LoF obs/exp: 17 / 25.1Missense obs/exp: 228 / 237.5Syn Z: -1.80
DN
0.7035th %ile
GOF
0.78top 25%
LOF
0.3164th %ile

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

GOFprediction above median
DNprediction 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

215 submitted variants in ClinVar

Classification Summary

Pathogenic76
Likely Pathogenic4
VUS113
Likely Benign3
76
Pathogenic
4
Likely Pathogenic
113
VUS
3
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
76
0
76
Likely Pathogenic
0
0
4
0
4
VUS
0
102
11
0
113
Likely Benign
0
3
0
0
3
Benign
0
0
0
0
0
Total0105910196

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

View in ClinVar →

Protein Context — Lollipop Plot

MICU3 · 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.

Search ClinicalTrials.gov →
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Top 2 results · since 2015Search PubMed ↗