GRPEL1

Chr 4

GrpE like 1, mitochondrial

Also known as: GrpE, HMGE, mt-GrpE#1

The GRPEL1 protein is an essential component of the PAM complex that controls mitochondrial HSP70 binding to substrate proteins during ATP-dependent protein import into the mitochondrial matrix. Mutations cause autosomal recessive mitochondrial disease with early infantile onset, characterized by severe developmental delay, seizures, hypotonia, and progressive neurodegeneration. The gene shows moderate constraint against loss-of-function variants, consistent with recessive inheritance where heterozygous carriers are typically unaffected.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
14
Pubs (1 yr)
84
P/LP submissions
0%
P/LP missense
0.78
LOEUF
DN
Mechanism· predicted
Clinical SummaryGRPEL1
Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.30) despite low pLI — interpret in context.
📋
ClinVar Variants
84 unique Pathogenic / Likely Pathogenic· 31 VUS of 123 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.78LOEUF
pLI 0.139
Z-score 2.04
OE 0.30 (0.140.78)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
-0.01Z-score
OE missense 1.00 (0.861.16)
122 obs / 121.8 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.30 (0.140.78)
00.351.4
Missense OE1.00 (0.861.16)
00.61.4
Synonymous OE0.98
01.21.6
LoF obs/exp: 3 / 10.0Missense obs/exp: 122 / 121.8Syn Z: 0.14
DN
0.6743th %ile
GOF
0.5170th %ile
LOF
0.3358th %ile

The highest-scoring mechanism for this gene is dominant-negative.

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

123 submitted variants in ClinVar

Classification Summary

Pathogenic80
Likely Pathogenic4
VUS31
Likely Benign1
80
Pathogenic
4
Likely Pathogenic
31
VUS
1
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
80
0
80
Likely Pathogenic
0
0
4
0
4
VUS
0
28
3
0
31
Likely Benign
0
1
0
0
1
Benign
0
0
0
0
0
Total029870116

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

View in ClinVar →

Protein Context — Lollipop Plot

GRPEL1 · 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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