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10 sources retrieved · Most recent: April 2026 · Index updated 14 days ago
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ATP6V1C1
ATPase H+ transporting V1 subunit C1
Chromosome 8 · 8q22.3
NCBI Gene: 528Ensembl: ENSG00000155097.13HGNC: HGNC:856UniProt: A0A024R9I0
68PubMed Papers
20Diseases
0Drugs
1Pathogenic Variants
FUNCTIONAL ROLE
Transporter
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
protein bindingextracellular exosomelysosomal membraneplasma membraneneurodegenerative diseaseAlzheimer diseaselysosomal storage diseaseParkinson disease
✦AI Summary

ATP6V1C1 encodes the C1 subunit of the vacuolar H+-ATPase (V-ATPase), a multisubunit enzyme complex responsible for proton transport and cellular acidification 1. The protein is essential for V-ATPase assembly and catalytic activity, maintaining pH homeostasis in intracellular compartments including lysosomes and autophagosomes 12. Mechanistically, ATP6V1C1 regulates lysosomal acidification and autophagic flux, with pathogenic variants causing gain-of-function effects that disrupt cellular homeostasis 1. The protein also influences cytoskeletal organization by co-localizing with F-actin and affecting filamentous actin arrangement 3. Disease relevance includes dominant mutations causing neurodevelopmental syndromes like DOORS syndrome through altered lysosomal function 1, and specific variants causing non-syndromic sensorineural hearing loss by impairing hair cell synaptic function and survival 2. Clinically, ATP6V1C1 is overexpressed in various cancers including oral squamous cell carcinoma and breast cancer, where it promotes metastasis and radiotherapy resistance 456. The gene serves as a potential therapeutic target, with natural compounds like veratramine showing anti-cancer effects through ATP6V1C1 inhibition 6. Additionally, ATP6V1C1 variants influence skull bone mineral density and cranial development 7.

Sources cited
1
ATP6V1C1 encodes V-ATPase C1 subunit and pathogenic variants cause gain-of-function effects disrupting lysosomal function in DOORS syndrome
PMID: 39210597
2
ATP6V1C1 variants cause non-syndromic hearing loss through impaired hair cell function and synaptic defects
PMID: 41539473
3
ATP6V1C1 co-localizes with F-actin and affects filamentous actin arrangement in breast cancer cells
PMID: 24454753
4
ATP6V1C1 is overexpressed in oral squamous cell carcinoma during later stages of carcinogenesis
PMID: 26984774
5
ATP6V1C1 promotes radiotherapy resistance in esophageal cancer by inhibiting autophagy
PMID: 33183740
6
ATP6V1C1 is upregulated in tumors and serves as an anti-cancer drug target, with veratramine showing inhibitory effects
PMID: 40384877
7
ATP6V1C1 variants influence skull bone mineral density and cranial development
PMID: 37402774
Disease Associationsⓘ20
neurodegenerative diseaseOpen Targets
0.56Moderate
Alzheimer diseaseOpen Targets
0.50Moderate
lysosomal storage diseaseOpen Targets
0.50Moderate
Parkinson diseaseOpen Targets
0.50Moderate
multiple sclerosisOpen Targets
0.50Moderate
DOORS syndromeOpen Targets
0.34Weak
neuroinflammatory disorderOpen Targets
0.27Weak
neoplasmOpen Targets
0.10Suggestive
breast cancerOpen Targets
0.08Suggestive
hepatocellular carcinomaOpen Targets
0.08Suggestive
cancerOpen Targets
0.06Suggestive
periodontitisOpen Targets
0.06Suggestive
goutOpen Targets
0.06Suggestive
acute kidney injuryOpen Targets
0.05Suggestive
chronic kidney diseaseOpen Targets
0.05Suggestive
esophageal squamous cell carcinomaOpen Targets
0.05Suggestive
hemoglobin D diseaseOpen Targets
0.04Suggestive
hemoglobin E diseaseOpen Targets
0.04Suggestive
Blackfan-Diamond anemiaOpen Targets
0.04Suggestive
monosomy 7 myelodysplasia and leukemia syndrome 1Open Targets
0.04Suggestive
Pathogenic Variants1
NM_001695.5(ATP6V1C1):c.865G>A (p.Glu289Lys)Pathogenic
DOORS syndrome
★☆☆☆2024→ Residue 289
View on ClinVar ↗
Related Genes
ATP6V1AProtein interaction100%ATP6V0CProtein interaction100%ATP6V1HProtein interaction100%ATP6V1FProtein interaction100%ATP6V0D1Protein interaction100%ATP6V0BProtein interaction100%
Tissue Expression6 tissues
Brain
100%
Heart
42%
Lung
33%
Ovary
32%
Liver
32%
Bone Marrow
22%
Gene Interaction Network
Click a node to explore
ATP6V1C1ATP6V1AATP6V0CATP6V1HATP6V1FATP6V0D1ATP6V0B
PROTEIN STRUCTURE
Preparing viewer…
PDB6WM2 · 3.10 Å · EM
View on RCSB ↗
Constraintⓘ
LOEUFⓘ
1.16LoF Tolerant
pLIⓘ
0.00Tolerant
Observed/Expected LoF0.87 [0.66–1.16]
RankingsWhere ATP6V1C1 stands among ~20K protein-coding genes
  • #6,878of 20,598
    Most Researched68
  • #5,009of 5,498
    Most Pathogenic Variants1
  • #12,141of 17,882
    Most Constrained (LOEUF)1.16
Genes detectedATP6V1C1
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
Integrated thermal proteome and thermal proximity co-aggregation profiling identifies ATP6V1C1 as a novel anti-cancer drug target.
PMID: 40384877
Int J Biol Sci · 2025
1.00
2
Dominantly acting variants in ATP6V1C1 and ATP6V1B2 cause a multisystem phenotypic spectrum by altering lysosomal and/or autophagosome function.
PMID: 39210597
HGG Adv · 2024
0.90
3
ATP6V1C1 deficiency impairs auditory and vestibular hair cell function and leads to sensorineural hearing loss in humans and mice.
PMID: 41539473
J Genet Genomics · 2026
0.80
4
Integrated multiomics analysis identifies potential biomarkers and therapeutic targets for autophagy associated AKI to CKD transition.
PMID: 40258914
Sci Rep · 2025
0.70
5
Expression of ATP6V1C1 during oral carcinogenesis.
PMID: 26984774
Biotech Histochem · 2016
0.60