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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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NDUFAF3
NADH:ubiquinone oxidoreductase complex assembly factor 3
Chromosome 3 Β· 3p21.31
NCBI Gene: 25915Ensembl: ENSG00000178057.16HGNC: HGNC:29918UniProt: A4FU71
57PubMed Papers
21Diseases
3Drugs
8Pathogenic Variants
FUNCTIONAL ROLE
Hub Gene
CLINICAL
FDA Approved TargetOMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
protein bindingmitochondrial respiratory chain complex I assemblymitochondrial inner membranenucleusmitochondrial complex I deficiencymitochondrial complex I deficiency, nuclear type 18mitochondrial complex I deficiency, nuclear type 1type 2 diabetes mellitus
✦AI Summary

NDUFAF3 is an essential mitochondrial assembly factor required for complex I (NADH:ubiquinone oxidoreductase) biogenesis. It functions as a structural component facilitating the assembly of complex I subunits, interacting directly with NDUFAF4 and other assembly proteins 1. NDUFAF3 coordinates the sequential assembly of functional modules, including the membrane arm and Q-module/P-module components, ensuring proper complex I maturation and respiratory supercomplex formation 2. Pathogenic NDUFAF3 variants cause mitochondrial complex I deficiency manifesting as autosomal recessive mitochondrial disease 3. Clinical presentations range from severe neonatal presentations with fatal outcomes and lactic acidosis 1 to more variable phenotypes including Leigh syndrome, cavitating leukoencephalopathy 4, progressive optic atrophy 5, and dystonia with basal ganglia abnormalities 2. Biochemically, NDUFAF3 deficiency results in reduced complex I activity, impaired assembly of early sub-complexes, and accumulation of intermediate assembly forms. Disease severity correlates with the degree of complex I dysfunction; assembly factor variants generally produce more variable phenotypes compared to core subunit defects 5. These findings establish NDUFAF3 as a critical determinant of mitochondrial respiratory function with broad neurological disease implications.

Sources cited
1
NDUFAF3 is an mitochondrial complex I assembly protein that interacts with NDUFAF4 and complex I subunits; mutations cause fatal neonatal mitochondrial disease
PMID: 19463981
2
NDUFAF3 variants cause complex I deficiency with variable phenotypes including neurodevelopmental disorder and dystonia; defects result in reduced CI activity and accumulation of early sub-assemblies
PMID: 37572574
3
NDUFAF3 is a complex I assembly factor; variants cause Leigh syndrome and link NDUFAF3 to specific steps in complex I assembly
PMID: 27986404
4
NDUFAF3 variants disrupt mitochondrial complex I assembly and associate with cavitating leukoencephalopathy and reduced complex I respiratory enzyme activity
PMID: 29344937
5
NDUFAF3 is a nuclear-encoded complex I assembly factor; biallelic variants cause isolated or syndromic optic atrophy and associate with autosomal recessive Leber Hereditary Optic Neuropathy phenotype
PMID: 41234160
Disease Associationsβ“˜21
mitochondrial complex I deficiencyOpen Targets
0.75Strong
mitochondrial complex I deficiency, nuclear type 18Open Targets
0.66Moderate
mitochondrial complex I deficiency, nuclear type 1Open Targets
0.64Moderate
type 2 diabetes mellitusOpen Targets
0.61Moderate
diabetes mellitusOpen Targets
0.61Moderate
mitochondrial diseaseOpen Targets
0.60Moderate
polycystic ovary syndromeOpen Targets
0.41Moderate
gestational diabetesOpen Targets
0.41Moderate
Insulin resistanceOpen Targets
0.41Moderate
obesityOpen Targets
0.40Weak
prediabetes syndromeOpen Targets
0.40Weak
metabolic syndromeOpen Targets
0.39Weak
type 1 diabetes mellitusOpen Targets
0.39Weak
Disorder of lipid metabolismOpen Targets
0.38Weak
agingOpen Targets
0.37Weak
prostate cancerOpen Targets
0.36Weak
COVID-19Open Targets
0.36Weak
abnormal glucose toleranceOpen Targets
0.36Weak
metabolic diseaseOpen Targets
0.35Weak
breast cancerOpen Targets
0.34Weak
Mitochondrial complex I deficiency, nuclear type 18UniProt
Pathogenic Variants8
NM_199069.2(NDUFAF3):c.447del (p.Cys150fs)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2020β†’ Residue 150
NM_199069.2(NDUFAF3):c.2T>C (p.Met1Thr)Pathogenic
Mitochondrial complex I deficiency, nuclear type 18|not provided
β˜…β˜†β˜†β˜†2020β†’ Residue 1
NM_199069.2(NDUFAF3):c.494C>T (p.Ala165Val)Likely pathogenic
Mitochondrial complex I deficiency, nuclear type 18
β˜…β˜†β˜†β˜†2019β†’ Residue 165
NM_199069.2(NDUFAF3):c.127C>T (p.Gln43Ter)Likely pathogenic
Mitochondrial complex I deficiency, nuclear type 18
β˜…β˜†β˜†β˜†β†’ Residue 43
NM_199069.2(NDUFAF3):c.77+1G>APathogenic
Mitochondrial complex I deficiency, nuclear type 18
β˜†β˜†β˜†β˜†2024
NM_199069.2(NDUFAF3):c.302G>A (p.Ser101Asn)Pathogenic
Mitochondrial complex I deficiency, nuclear type 18
β˜†β˜†β˜†β˜†2023β†’ Residue 101
NM_199069.2(NDUFAF3):c.365G>C (p.Arg122Pro)Pathogenic
Mitochondrial complex I deficiency, nuclear type 18
β˜†β˜†β˜†β˜†2009β†’ Residue 122
NM_199069.2(NDUFAF3):c.229G>C (p.Gly77Arg)Pathogenic
Mitochondrial complex I deficiency, nuclear type 18
β˜†β˜†β˜†β˜†2009β†’ Residue 77
View on ClinVar β†—
Drug Targets3
ME-344Phase I/II
Mitochondrial complex I (NADH dehydrogenase) inhibitor
breast cancer
METFORMINApproved
Mitochondrial complex I (NADH dehydrogenase) inhibitor
diabetes mellitus
METFORMIN HYDROCHLORIDEApproved
Mitochondrial complex I (NADH dehydrogenase) inhibitor
type 2 diabetes mellitus
Related Genes
NDUFAF8Shared pathway100%DMAC1Shared pathway100%LYRM2Shared pathway100%NDUFAF5Protein interaction100%SURF1Protein interaction100%NDUFB5Protein interaction100%
Tissue Expression6 tissues
Heart
100%
Liver
73%
Brain
56%
Lung
55%
Ovary
53%
Bone Marrow
47%
Gene Interaction Network
Click a node to explore
NDUFAF3NDUFAF8DMAC1LYRM2NDUFAF5SURF1NDUFB5
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q9BU61
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
1.22LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.85 [0.61–1.22]
RankingsWhere NDUFAF3 stands among ~20K protein-coding genes
  • #7,992of 20,598
    Most Researched57
  • #656of 1,025
    FDA-Approved Drug Targets2
  • #3,082of 5,498
    Most Pathogenic Variants8
  • #12,841of 17,882
    Most Constrained (LOEUF)1.22
Genes detectedNDUFAF3
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Recessive variants in mitochondrial Complex I nuclear subunits are an underrated cause of optic atrophy.
PMID: 41234160
Brain Β· 2025
1.00
2
Expanding the phenotypic and biochemical spectrum of NDUFAF3-related mitochondrial disease.
PMID: 37572574
Mol Genet Metab Β· 2023
0.90
3
Integrative analysis of the human cis-antisense gene pairs, miRNAs and their transcription regulation patterns.
PMID: 19906709
Nucleic Acids Res Β· 2010
0.80
4
Integrative genetics and multiomics analysis reveal mechanisms and therapeutic targets in vitiligo highlighting JAK STAT pathway regulation of CTSS.
PMID: 39824912
Sci Rep Β· 2025
0.70
5
Mutations in mitochondrial complex I assembly factor NDUFAF3 cause Leigh syndrome.
PMID: 27986404
Mol Genet Metab Β· 2017
0.60