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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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SURF1
SURF1 cytochrome c oxidase assembly factor
Chromosome 9 Β· 9q34.2
NCBI Gene: 6834Ensembl: ENSG00000148290.10HGNC: HGNC:11474UniProt: A0A087WYS9
64PubMed Papers
22Diseases
0Drugs
173Pathogenic Variants
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
mitochondrial respiratory chain complex IV assemblyprotein bindingmitochondrionmitochondrial inner membraneLeigh syndromeCharcot-Marie-Tooth disease type 4KSURF1-related Charcot-Marie-Tooth disease type 4mitochondrial complex IV deficiency, nuclear type 22
✦AI Summary

SURF1 encodes a cytochrome c oxidase (COX) assembly factor essential for complex IV biogenesis and stability in the mitochondrial inner membrane 1. As a component of the MITRAC complex, SURF1 regulates the assembly of the electron transport chain's terminal oxidase, which catalyzes oxygen reduction during aerobic respiration 2. SURF1 mutations impair COX activity, reducing it to approximately 32% of normal levels 3, compromising mitochondrial oxidative phosphorylation and cellular energy production. Pathogenic SURF1 variants cause Leigh syndrome, a severe pediatric neurodegenerative disease characterized by symmetric basal ganglia and brainstem lesions, developmental regression, elevated serum lactate, and early mortality (≀50% three-year survival) 4. SURF1 mutations also associate with Charcot-Marie-Tooth disease type 4K and nuclear-encoded complex IV deficiency 1. Over 60 different SURF1 mutations have been identified, with frameshift and nonsense variants causing more severe phenotypes 3. Clinical management includes ketogenic diet and coenzyme Q supplementation, though results remain inconsistent 1. Emerging gene therapy approaches utilizing improved AAV9 vectors show promise for restoring COX activity without cytotoxicity 5. COX activity measurements represent a potential biomarker for disease severity and treatment monitoring 3.

Sources cited
1
SURF1 encodes assembly factor for COX stability; mutations cause Leigh syndrome with infancy onset, grave prognosis, basal ganglia/brainstem involvement, and relationship between genotype and phenotype; treatment options including ketogenic diet and coenzyme Q
PMID: 34943053
2
SURF1 deficiency reduces COX activity to ~32% of controls; bi-allelic variants, brainstem abnormalities, motor regression common; nonsense/frameshift variants correlate with severe phenotypes and MRI abnormalities; COX activity as treatment biomarker
PMID: 39632678
3
SURF1 among most frequent genes causing Leigh syndrome; associated with poorest survival (≀50% 3-year survival); elevated serum lactate, global developmental delay, developmental regression most frequent features
PMID: 35094435
4
SURF1 gene defect causes nuclear-encoded COX deficiency associated with Leigh syndrome; SURF1, COX10, and SCO2 involved in COX assembly with different phenotypic presentations
PMID: 11044474
5
AAV9-based gene therapy for SURF1 Leigh syndrome improves complex IV activity; vector design optimization can achieve efficacy with reduced cytotoxicity
PMID: 40893166
6
Over 60 different SURF1 mutations identified in Leigh syndrome; atypical courses with milder symptoms possible; anti-epileptic drugs like valproate should be avoided
PMID: 34943053
Disease Associationsβ“˜22
Leigh syndromeOpen Targets
0.80Strong
Charcot-Marie-Tooth disease type 4KOpen Targets
0.76Strong
SURF1-related Charcot-Marie-Tooth disease type 4Open Targets
0.69Moderate
mitochondrial complex IV deficiency, nuclear type 22Open Targets
0.68Moderate
Isolated cytochrome C oxidase deficiencyOpen Targets
0.66Moderate
mitochondrial diseaseOpen Targets
0.65Moderate
leigh syndrome due to mitochondrial complex iv deficiencyOpen Targets
0.57Moderate
mitochondrial complex IV deficiency, nuclear-typeOpen Targets
0.53Moderate
genetic disorderOpen Targets
0.52Moderate
cerebellar ataxiaOpen Targets
0.41Moderate
Abnormal pyramidal signOpen Targets
0.41Moderate
DysarthriaOpen Targets
0.41Moderate
Muscle weaknessOpen Targets
0.41Moderate
fatal infantile encephalocardiomyopathyOpen Targets
0.34Weak
asthmaOpen Targets
0.04Suggestive
rhabdomyolysisOpen Targets
0.04Suggestive
cancerOpen Targets
0.03Suggestive
myocardial infarctionOpen Targets
0.03Suggestive
COVID-19Open Targets
0.02Suggestive
hyperinsulinemic hypoglycemia, familial, 4Open Targets
0.02Suggestive
Charcot-Marie-Tooth disease, demyelinating, type 4KUniProt
Mitochondrial complex IV deficiency, nuclear type 1UniProt
Pathogenic Variants173
NM_003172.4(SURF1):c.845_846del (p.Ser282fs)Pathogenic
Mitochondrial complex IV deficiency, nuclear type 1|Leigh syndrome|not provided|Charcot-Marie-Tooth disease type 4K;Leigh syndrome due to mitochondrial complex IV deficiency|Inborn genetic diseases|Abnormal pyramidal sign;Muscle weakness;Dysarthria;Cerebellar ataxia|See cases|SURF1-related disorder|Charcot-Marie-Tooth disease type 4K;Mitochondrial complex IV deficiency, nuclear type 1
β˜…β˜…β˜†β˜†2026β†’ Residue 282
NM_003172.4(SURF1):c.688C>T (p.Arg230Ter)Pathogenic
not provided|Leigh syndrome|Mitochondrial complex IV deficiency, nuclear type 1;Charcot-Marie-Tooth disease type 4K
β˜…β˜…β˜†β˜†2026β†’ Residue 230
NM_003172.4(SURF1):c.187C>T (p.Gln63Ter)Pathogenic
Mitochondrial complex IV deficiency, nuclear type 1|Leigh syndrome
β˜…β˜…β˜†β˜†2026β†’ Residue 63
NM_003172.4(SURF1):c.183_186del (p.Leu62fs)Pathogenic
Leigh syndrome
β˜…β˜…β˜†β˜†2026β†’ Residue 62
NM_003172.4(SURF1):c.752-3C>GLikely pathogenic
Leigh syndrome
β˜…β˜…β˜†β˜†2026
NM_003172.4(SURF1):c.269T>C (p.Leu90Pro)Pathogenic
not provided|Leigh syndrome|Inborn genetic diseases
β˜…β˜…β˜†β˜†2026β†’ Residue 90
NM_003172.4(SURF1):c.574_575insCTGC (p.Arg192fs)Pathogenic
not provided|Leigh syndrome|Mitochondrial complex IV deficiency, nuclear type 1|Inborn genetic diseases
β˜…β˜…β˜†β˜†2026β†’ Residue 192
NM_003172.4(SURF1):c.491C>T (p.Thr164Ile)Likely pathogenic
not provided|Leigh syndrome|Mitochondrial complex IV deficiency, nuclear type 1
β˜…β˜…β˜†β˜†2026β†’ Residue 164
NM_003172.4(SURF1):c.552del (p.Lys185fs)Pathogenic
Leigh syndrome|Mitochondrial complex IV deficiency, nuclear type 1|not provided|Mitochondrial complex IV deficiency, nuclear type 1;Charcot-Marie-Tooth disease type 4K
β˜…β˜…β˜†β˜†2026β†’ Residue 185
NM_003172.4(SURF1):c.311_312insA (p.Leu105fs)Pathogenic
not provided|Charcot-Marie-Tooth disease type 4K|See cases|Mitochondrial complex IV deficiency, nuclear type 1|Leigh syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 105
NM_003172.4(SURF1):c.792_793del (p.Arg264fs)Pathogenic
Mitochondrial complex IV deficiency, nuclear type 1|not provided|Leigh syndrome|Charcot-Marie-Tooth disease type 4K|Inborn genetic diseases
β˜…β˜…β˜†β˜†2025β†’ Residue 264
NM_003172.4(SURF1):c.555_556del (p.Lys186fs)Pathogenic
Leigh syndrome|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 186
NM_003172.4(SURF1):c.820_824dup (p.Val276fs)Pathogenic
Leigh syndrome|Charcot-Marie-Tooth disease type 4K;Mitochondrial complex IV deficiency, nuclear type 1
β˜…β˜…β˜†β˜†2025β†’ Residue 276
NM_003172.4(SURF1):c.575G>A (p.Arg192Gln)Pathogenic
Leigh syndrome|Mitochondrial complex IV deficiency, nuclear type 1|Charcot-Marie-Tooth disease type 4K;Mitochondrial complex IV deficiency, nuclear type 1
β˜…β˜…β˜†β˜†2025β†’ Residue 192
NM_003172.4(SURF1):c.312_321delinsAT (p.Pro104_Leu105insTer)Pathogenic
Mitochondrial complex IV deficiency, nuclear type 1|not provided|Leigh syndrome|Charcot-Marie-Tooth disease type 4K;Mitochondrial complex IV deficiency, nuclear type 1|SURF1-related disorder
β˜…β˜…β˜†β˜†2025β†’ Residue 104
NM_003172.4(SURF1):c.-11_13del (p.Met1_Ala5del)Pathogenic
not provided|Leigh syndrome|Charcot-Marie-Tooth disease type 4K;Mitochondrial complex IV deficiency, nuclear type 1|Mitochondrial complex IV deficiency, nuclear type 1
β˜…β˜…β˜†β˜†2025β†’ Residue 1
NM_003172.4(SURF1):c.752-1G>CPathogenic
Leigh syndrome|not provided
β˜…β˜…β˜†β˜†2025
NM_003172.4(SURF1):c.751+1G>APathogenic
Leigh syndrome|not provided
β˜…β˜…β˜†β˜†2025
NM_003172.4(SURF1):c.809_826dup (p.Glu270_Ile275dup)Pathogenic
Leigh syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 270
NM_003172.4(SURF1):c.586C>T (p.Gln196Ter)Pathogenic
Leigh syndrome|not provided|SURF1-related disorder|Mitochondrial complex IV deficiency, nuclear type 1;Charcot-Marie-Tooth disease type 4K|Hepatocellular carcinoma
β˜…β˜…β˜†β˜†2025β†’ Residue 196
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Related Genes
NDUFAF3Protein interaction100%NDUFV1Protein interaction100%BCS1LProtein interaction99%COX10Protein interaction99%COX11Protein interaction99%COX15Protein interaction99%
Tissue Expression6 tissues
Liver
100%
Heart
60%
Brain
50%
Lung
45%
Ovary
38%
Bone Marrow
34%
Gene Interaction Network
Click a node to explore
SURF1NDUFAF3NDUFV1BCS1LCOX10COX11COX15
PROTEIN STRUCTURE
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AlphaFoldAI-predicted Β· UniProt Q15526
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
1.56LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF1.21 [0.95–1.56]
RankingsWhere SURF1 stands among ~20K protein-coding genes
  • #7,320of 20,598
    Most Researched64
  • #424of 5,498
    Most Pathogenic Variants173 Β· top 10%
  • #15,477of 17,882
    Most Constrained (LOEUF)1.56
Genes detectedSURF1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Leigh Syndrome: A Study of 209 Patients at the Beijing Children's Hospital.
PMID: 35094435
Ann Neurol Β· 2022
1.00
2
A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort.
PMID: 34602496
J Neuromuscul Dis Β· 2022
0.90
3
Clinical Diagnosis and Treatment of Leigh Syndrome Based on
PMID: 34943053
Antioxidants (Basel) Β· 2021
0.80
4
SURF1 Deficiency: Expanding on Disease Phenotype and Assessing Disease Burden by Describing Clinical and Biochemical Phenotype.
PMID: 39632678
Am J Med Genet A Β· 2025
0.70
5
Improved AAV9-based gene therapy design for
PMID: 40893166
Mol Ther Methods Clin Dev Β· 2025
0.60