chrM-4332-G-A

Variant summary

Our verdict is Uncertain significance. The variant received 3 ACMG points: 3P and 0B. PS3_SupportingPP3PM2_Supporting

This summary comes from the ClinGen Evidence Repository: The m.4332G>A variant in MT-TQ has been reported in one individual from one family to date (PMID:11171912), in a man with an atypical presentation of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS). This individual had hearing loss beginning in his 20s and a stroke-like episode in his 40s. Muscle biopsy showed ragged red fibers and COX-deficient fibers, along with decreased activity of respiratory chain complex IV. The variant was present at 81% in muscle and undetectable in fibroblasts and skin. While the variant was absent in blood from his mother and brother, it was also undetectable in the proband’s blood, therefore it is unclear if this was a de novo variant. There are no large families reported in the medical literature to consider for evidence of segregation. Of note, there is another individual reported with MELAS and this variant, however insufficient clinical details were provided to include this case for consideration for this variant curation (PMID:20064630). The computational predictor MitoTIP suggests this variant impacts the function of this tRNA (81st percentile) as does HmtVar with a score of 0.7 (PP3). This variant is absent in the GenBank dataset, Helix dataset, and gnomAD v3.1.2 (PM2_supporting). Single fiber testing showed higher levels of heteroplasmy in COX-negative fibers (mean 99.3% and ranging from 98.8-99.7%, n=13) than in COX-positive fibers (mean 70.5% and ranging from 46.5-94.5%, n=11; p=0.002; PMID:11335700; PS3_supporting). In summary, this variant meets criteria to be classified as uncertain significance for primary mitochondrial disease inherited in a mitochondrial manner. This classification was approved by the NICHD/NINDS U24 ClinGen Mitochondrial Disease Variant Curation Expert Panel on January 23, 2023. Mitochondrial DNA-specific ACMG/AMP criteria applied (PMID:32906214): PM2_supporting, PP3, PS3_supporting. LINK:https://erepo.genome.network/evrepo/ui/classification/CA254844/MONDO:0044970/014

Frequency

Mitomap GenBank:
Absent

Consequence

TRNQ
unassigned_transcript_4791 synonymous

Scores

Mitotip
Pathogenic
17

Clinical Significance

Uncertain significance reviewed by expert panel P:2U:1O:1
Encephalopathy-/-MELAS

Conservation

PhyloP100: 3.85

Publications

3 publications found
Variant links:
Genes affected
TRNQ (HGNC:7495): (mitochondrially encoded tRNA glutamine)
MT-ND2 (HGNC:7456): (mitochondrially encoded NADH dehydrogenase 2) Enables NADH dehydrogenase (ubiquinone) activity. Involved in mitochondrial electron transport, NADH to ubiquinone and mitochondrial respiratory chain complex I assembly. Part of mitochondrial respiratory chain complex I. Implicated in Leber hereditary optic neuropathy; multiple sclerosis; myocardial infarction; neurodegenerative disease (multiple); and urinary bladder cancer. [provided by Alliance of Genome Resources, Apr 2022]
MT-ND1 (HGNC:7455): (mitochondrially encoded NADH dehydrogenase 1) Enables NADH dehydrogenase (ubiquinone) activity. Involved in mitochondrial electron transport, NADH to ubiquinone and mitochondrial respiratory chain complex I assembly. Located in mitochondrial membrane. Part of mitochondrial respiratory chain complex I. Implicated in several diseases, including MELAS syndrome; neurodegenerative disease (multiple); optic nerve disease (multiple); toxic shock syndrome; and type 2 diabetes mellitus. Biomarker of Alzheimer's disease; Parkinson's disease; and multiple sclerosis. [provided by Alliance of Genome Resources, Apr 2022]
TRNM (HGNC:7492): (mitochondrially encoded tRNA methionine)
TRNI (HGNC:7488): (mitochondrially encoded tRNA isoleucine)
TRNI Gene-Disease associations (from GenCC):
  • mitochondrial disease
    Inheritance: Mitochondrial Classification: DEFINITIVE Submitted by: ClinGen
  • Leigh syndrome
    Inheritance: Mitochondrial Classification: LIMITED Submitted by: ClinGen

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ACMG classification

Classification was made for transcript

Our verdict: Uncertain_significance. The variant received 3 ACMG points.

PS3
For more information check the summary or visit ClinGen Evidence Repository.
PM2
For more information check the summary or visit ClinGen Evidence Repository.
PP3
For more information check the summary or visit ClinGen Evidence Repository.

Transcripts

RefSeq

Gene Transcript HGVSc HGVSp Effect Exon rank MANE Protein UniProt
TRNQunassigned_transcript_4791 c.69C>T p.Ser23Ser synonymous_variant Exon 1 of 1
ND2unassigned_transcript_4793 c.-138G>A upstream_gene_variant
TRNMunassigned_transcript_4792 c.-70G>A upstream_gene_variant

Ensembl

Gene Transcript HGVSc HGVSp Effect Exon rank TSL MANE Protein Appris UniProt
MT-ND2ENST00000361453.3 linkc.-138G>A upstream_gene_variant 6 ENSP00000355046.4
MT-ND1ENST00000361390.2 linkc.*70G>A downstream_gene_variant 6 ENSP00000354687.2

Frequencies

Mitomap GenBank
The variant is not present, suggesting it is rare.
Gnomad homoplasmic
AF:
0.0
AC:
0
AN:
56431
Gnomad heteroplasmic
AF:
0.000018
AC:
1
AN:
56431

Mitomap

Disease(s): Encephalopathy-/-MELAS
Status: Cfrm-[VUS*]
Publication(s): 11335700

ClinVar

Significance: Uncertain significance
Submissions summary: Pathogenic:2Uncertain:1Other:1
Revision: reviewed by expert panel
LINK: link

Submissions by phenotype

MELAS syndrome Pathogenic:1Other:1
Feb 13, 2001
OMIM
Significance:Pathogenic
Review Status:no assertion criteria provided
Collection Method:literature only

GeneReviews
Significance:not provided
Review Status:no classification provided
Collection Method:literature only

MT-TQ-related myopathy Pathogenic:1
Jul 30, 2024
Undiagnosed Diseases Network, NIH
Significance:Pathogenic
Review Status:criteria provided, single submitter
Collection Method:clinical testing

Mitochondrial disease Uncertain:1
Jan 23, 2023
ClinGen Mitochondrial Disease Nuclear and Mitochondrial Variant Curation Expert Panel, ClinGen
Significance:Uncertain significance
Review Status:reviewed by expert panel
Collection Method:curation

The m.4332G>A variant in MT-TQ has been reported in one individual from one family to date (PMID: 11171912), in a man with an atypical presentation of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS). This individual had hearing loss beginning in his 20s and a stroke-like episode in his 40s. Muscle biopsy showed ragged red fibers and COX-deficient fibers, along with decreased activity of respiratory chain complex IV. The variant was present at 81% in muscle and undetectable in fibroblasts and skin. While the variant was absent in blood from his mother and brother, it was also undetectable in the proband’s blood, therefore it is unclear if this was a de novo variant. There are no large families reported in the medical literature to consider for evidence of segregation. Of note, there is another individual reported with MELAS and this variant, however insufficient clinical details were provided to include this case for consideration for this variant curation (PMID: 20064630). The computational predictor MitoTIP suggests this variant impacts the function of this tRNA (81st percentile) as does HmtVar with a score of 0.7 (PP3). This variant is absent in the GenBank dataset, Helix dataset, and gnomAD v3.1.2 (PM2_supporting). Single fiber testing showed higher levels of heteroplasmy in COX-negative fibers (mean 99.3% and ranging from 98.8-99.7%, n=13) than in COX-positive fibers (mean 70.5% and ranging from 46.5-94.5%, n=11; p=0.002; PMID: 11335700; PS3_supporting). In summary, this variant meets criteria to be classified as uncertain significance for primary mitochondrial disease inherited in a mitochondrial manner. This classification was approved by the NICHD/NINDS U24 ClinGen Mitochondrial Disease Variant Curation Expert Panel on January 23, 2023. Mitochondrial DNA-specific ACMG/AMP criteria applied (PMID: 32906214): PM2_supporting, PP3, PS3_supporting.

Computational scores

Source: dbNSFP v4.3

Name
Calibrated prediction
Score
Prediction
Mitotip
Pathogenic
17
Hmtvar
Pathogenic
0.70
PhyloP100
3.8

Publications

Other links and lift over

dbSNP: rs199476141; hg19: chrM-4333; API