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8 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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ATP5PO
ATP synthase peripheral stalk subunit OSCP
Chromosome 21 Β· 21q22.11
NCBI Gene: 539Ensembl: ENSG00000241837.8HGNC: HGNC:850UniProt: P48047
198PubMed Papers
21Diseases
0Drugs
3Pathogenic Variants
FUNCTIONAL ROLE
Hub GeneTransporter
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
proton motive force-driven mitochondrial ATP synthesisproton-transporting ATP synthase activity, rotational mechanismprotein bindingmitochondrionmitochondrial complex V (ATP synthase) deficiency, nuclear type 7Severe global developmental delayDysphagiaSeizure
✦AI Summary

ATP5PO encodes the oligomycin sensitivity-conferring protein (OSCP), a critical peripheral stalk subunit of mitochondrial ATP synthase (Complex V) 1. This protein stabilizes the peripheral stalk that links the catalytic F1 head domain to the membrane F0 domain, acting as a stator to maintain proper positioning of the catalytic core during ATP synthesis coupled to proton translocation 2. ATP5PO also functions as a key regulator of the mitochondrial permeability transition pore (mPTP) 3. Biallelic variants in ATP5PO cause severe mitochondrial complex V deficiency, manifesting as infantile-onset Leigh syndrome characterized by hypotonia, developmental delay, hypertrophic cardiomyopathy, progressive epilepsy, cerebral atrophy, and often early lethality 14. A homozygous splice variant (c.87+3A>G) produces a non-functional protein lacking exon 2, leading to reduced ATP5PO protein levels, defective complex V assembly, and markedly reduced oxidative phosphorylation 1. Heterozygous de novo mutations also cause variable neurologic phenotypes including dystonia and developmental delay 5. ATP5PO overexpression paradoxically reduces ATP5PO protein levels and impairs ENS development, linking ATP5PO dosage balance to Hirschsprung disease risk in Down Syndrome patients 6. Additionally, ATP5PO glutathionylation regulates mPTP function and represents a therapeutic vulnerability in acute myeloid leukemia 3.

Sources cited
1
ATP5PO is a subunit of the F0 domain and peripheral stalk of ATP synthase, linking F1 and F0 domains
PMID: 37244256
2
Homozygous splice variant in ATP5PO causes Leigh syndrome with defective complex V assembly and function
PMID: 35621276
3
ATP5PO homozygous splice variant (c.87+3A>G) causes complex V deficiency with reduced ATPase activity in fibroblasts
PMID: 40913360
4
Heterozygous and compound heterozygous ATP5PO variants cause variable neurologic phenotypes with reduced ATPase amounts
PMID: 34954817
5
ATP5PO overexpression reduces ENS cells and impairs neuronal differentiation; balanced ATP5PO expression required for normal ENS development
PMID: 38128843
6
ATP5PO is a key regulator of mPTP opening and critical GLRX2 target; ATP5PO glutathionylation regulates mPTP function
PMID: 41237364
Disease Associationsβ“˜21
mitochondrial complex V (ATP synthase) deficiency, nuclear type 7Open Targets
0.45Moderate
DysphagiaOpen Targets
0.43Moderate
microcephalyOpen Targets
0.43Moderate
SeizureOpen Targets
0.43Moderate
Severe global developmental delayOpen Targets
0.43Moderate
COVID-19Open Targets
0.40Moderate
mitochondrial diseaseOpen Targets
0.37Weak
goutOpen Targets
0.28Weak
Leigh syndromeOpen Targets
0.27Weak
clonal hematopoiesisOpen Targets
0.24Weak
atrial fibrillationOpen Targets
0.22Weak
central nervous system infectionOpen Targets
0.21Weak
major depressive disorderOpen Targets
0.17Weak
MODYOpen Targets
0.08Suggestive
Alzheimer diseaseOpen Targets
0.07Suggestive
type 1 diabetes mellitusOpen Targets
0.07Suggestive
posterior cortical atrophyOpen Targets
0.05Suggestive
maturity-onset diabetes of the young type 4Open Targets
0.05Suggestive
diabetes mellitus, transient neonatal, 2Open Targets
0.05Suggestive
maturity-onset diabetes of the young type 6Open Targets
0.05Suggestive
Mitochondrial complex V deficiency, nuclear type 7UniProt
Pathogenic Variants3
NM_001697.3(ATP5PO):c.87+3A>GPathogenic
ATP5PO-related disorder|Leigh syndrome|Mitochondrial complex V (ATP synthase) deficiency, nuclear type 7
β˜…β˜…β˜†β˜†2020
NM_001697.3(ATP5PO):c.329-20A>GPathogenic
Severe global developmental delay;Dysphagia;Seizure;Microcephaly|Mitochondrial complex V (ATP synthase) deficiency, nuclear type 7
β˜…β˜†β˜†β˜†2021
NM_001697.3(ATP5PO):c.34C>T (p.Gln12Ter)Pathogenic
Severe global developmental delay;Dysphagia;Seizure;Microcephaly|Mitochondrial complex V (ATP synthase) deficiency, nuclear type 7
β˜…β˜†β˜†β˜†2021β†’ Residue 12
View on ClinVar β†—
Related Genes
ATP5F1EProtein interaction100%ATP5MC1Protein interaction100%ATP6V1AProtein interaction100%ATP6V1B1Protein interaction100%ATP6V1B2Protein interaction100%ATP6V0CProtein interaction100%
Tissue Expression6 tissues
Heart
100%
Brain
35%
Liver
29%
Bone Marrow
23%
Ovary
23%
Lung
18%
Gene Interaction Network
Click a node to explore
ATP5POATP5F1EATP5MC1ATP6V1AATP6V1B1ATP6V1B2ATP6V0C
PROTEIN STRUCTURE
Preparing viewer…
PDB8H9E Β· 2.53 Γ… Β· EM
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.87LoF Tolerant
pLIβ“˜
0.01Tolerant
Observed/Expected LoF0.55 [0.35–0.87]
RankingsWhere ATP5PO stands among ~20K protein-coding genes
  • #2,136of 20,598
    Most Researched198 Β· top quartile
  • #3,938of 5,498
    Most Pathogenic Variants3
  • #7,663of 17,882
    Most Constrained (LOEUF)0.87
Genes detectedATP5PO
Sources retrieved8 papers
Response timeβ€”
πŸ“„ Sources
8β–Ό
1
Variants in Mitochondrial ATP Synthase Cause Variable Neurologic Phenotypes.
PMID: 34954817
Ann Neurol Β· 2022
1.00
2
ATP5PO levels regulate enteric nervous system development in zebrafish, linking Hirschsprung disease to Down Syndrome.
PMID: 38128843
Biochim Biophys Acta Mol Basis Dis Β· 2024
0.88
3
Glutaredoxin 2 is essential for AML survival through mitochondrial permeability transition pore regulation.
PMID: 41237364
Blood Β· 2026
0.75
4
Transcriptome research of human amniocytes identifies hub genes associated with developmental dysplasia in down syndrome.
PMID: 38095646
Aging (Albany NY) Β· 2023
0.63
5
A homozygous splice variant in ATP5PO, disrupts mitochondrial complex V function and causes Leigh syndrome in two unrelated families.
PMID: 35621276
J Inherit Metab Dis Β· 2022
0.50