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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
TCIRG1
T cell immune regulator 1, ATPase H+ transporting V0 subunit a3
Chromosome 11 Β· 11q13.2
NCBI Gene: 10312Ensembl: ENSG00000110719.11HGNC: HGNC:11647UniProt: Q13488
96PubMed Papers
21Diseases
0Drugs
278Pathogenic Variants
FUNCTIONAL ROLE
Transporter
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
protein bindinglysosomal membraneapical plasma membranevacuolar proton-transporting V-type ATPase complexAutosomal recessive malignant osteopetrosisautosomal recessive osteopetrosis 1osteopetrosisdysosteosclerosis
✦AI Summary

TCIRG1 encodes the a3 subunit of vacuolar H+-ATPase (V-ATPase), a multisubunit proton pump complex essential for acidifying intracellular compartments 1. As an integral component of the V0 membrane domain, TCIRG1 is primarily localized to lysosomes in mammalian cells, where it regulates late phagosome-lysosomal fusion and maintains lysosomal acidification 1. Defective TCIRG1 impairs phagosome maturation, autophagy, and cellular degradation pathways critical for immune cell function 1. Biallelic TCIRG1 mutations cause autosomal recessive osteopetrosis (ARO), affecting >50% of cases, due to osteoclast dysfunction and impaired bone resorption 23. The disease manifests as dense, fragile bone with severe complications including hepatosplenomegaly, thrombocytopenia, visual/hearing impairment, and infections 4. Heterozygous TCIRG1 variants also cause severe congenital neutropenia (SCN), with reduced TCIRG1 protein levels in affected individuals 5. Computational studies identify highly conserved protein domains critical for V-ATPase function; mutations in these regions substantially destabilize protein structure and impair catalytic activity 6. Gene therapy approaches using lentiviral vectors demonstrate post-transcriptional regulation preventing pathological overexpression, offering potential therapeutic strategies for osteopetrosis 7.

Sources cited
1
TCIRG1/Atp6v0a3 localizes to lysosomes and regulates late phagosome-lysosomal fusion; defects impair acidification and autophagy in microglia
PMID: 38873931
2
Biallelic TCIRG1 mutations cause >50% of autosomal recessive osteopetrosis cases through V-ATPase dysfunction
PMID: 22231430
3
TCIRG1 mutations identified in osteopetrosis patients include novel homozygous and compound heterozygous variants
PMID: 30898715
4
Malignant infantile osteopetrosis presents with hepatosplenomegaly, thrombocytopenia, sensory impairment, and infections caused by TCIRG1 mutations
PMID: 31859718
5
Heterozygous TCIRG1 variants cause severe congenital neutropenia with reduced TCIRG1 protein levels
PMID: 24753205
6
Disease-associated TCIRG1 mutations destabilize protein structure in V-ATPase I domain, impairing ATPase effectiveness
PMID: 35573728
7
Lentiviral vector-mediated TCIRG1 expression is post-transcriptionally regulated in osteoclasts and restores bone resorptive function
PMID: 27541021
Disease Associationsβ“˜21
Autosomal recessive malignant osteopetrosisOpen Targets
0.77Strong
autosomal recessive osteopetrosis 1Open Targets
0.75Strong
osteopetrosisOpen Targets
0.55Moderate
dysosteosclerosisOpen Targets
0.44Moderate
autosomal recessive osteopetrosisOpen Targets
0.44Moderate
genetic disorderOpen Targets
0.42Moderate
Intermediate osteopetrosisOpen Targets
0.38Weak
neutropenia, severe congenital, 1, autosomal dominantOpen Targets
0.38Weak
severe congenital neutropeniaOpen Targets
0.38Weak
autosomal dominant severe congenital neutropeniaOpen Targets
0.37Weak
neurodegenerative diseaseOpen Targets
0.34Weak
secondary malignant neoplasmOpen Targets
0.33Weak
neutropeniaOpen Targets
0.31Weak
Decreased total neutrophil countOpen Targets
0.31Weak
Abnormality of the skeletal systemOpen Targets
0.27Weak
chorea-acanthocytosisOpen Targets
0.27Weak
ChoreoacanthocytosisOpen Targets
0.27Weak
hypertensionOpen Targets
0.15Weak
color vision disorderOpen Targets
0.11Weak
nonpapillary renal cell carcinomaOpen Targets
0.09Suggestive
Osteopetrosis, autosomal recessive 1UniProt
Pathogenic Variants278
NM_006019.4(TCIRG1):c.117+4A>TPathogenic
Autosomal recessive osteopetrosis 1|not provided|Inborn genetic diseases|Autosomal recessive osteopetrosis|TCIRG1-related disorder
β˜…β˜…β˜†β˜†2026
NM_006019.4(TCIRG1):c.117+5G>ALikely pathogenic
not provided|Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2026
NM_006019.4(TCIRG1):c.1674-1G>APathogenic
Autosomal recessive osteopetrosis 1|not provided|Osteopetrosis|TCIRG1-related disorder|Clear cell carcinoma of kidney
β˜…β˜…β˜†β˜†2026
NM_006019.4(TCIRG1):c.979C>T (p.Arg327Ter)Pathogenic
Autosomal recessive osteopetrosis 1|not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 327
NM_006019.4(TCIRG1):c.702del (p.Ile235fs)Pathogenic
Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2026β†’ Residue 235
NM_006019.4(TCIRG1):c.862_866delinsG (p.Leu288fs)Pathogenic
not provided|Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2026β†’ Residue 288
NM_006019.4(TCIRG1):c.1384_1386del (p.Asn462del)Likely pathogenic
Autosomal recessive osteopetrosis 1|not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 462
NM_006019.4(TCIRG1):c.1891del (p.Val631fs)Pathogenic
Autosomal recessive osteopetrosis 1|not provided|TCIRG1-related disorder
β˜…β˜…β˜†β˜†2026β†’ Residue 631
NM_006019.4(TCIRG1):c.1967del (p.Leu656fs)Pathogenic
Autosomal recessive osteopetrosis 1|not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 656
NM_006019.4(TCIRG1):c.2161_2163del (p.Ile721del)Pathogenic
not provided|Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2026β†’ Residue 721
NM_006019.4(TCIRG1):c.117+1G>APathogenic
Autosomal recessive osteopetrosis 1|not provided
β˜…β˜…β˜†β˜†2026
NM_006019.4(TCIRG1):c.2236+1G>APathogenic
Autosomal recessive osteopetrosis 1|not provided
β˜…β˜…β˜†β˜†2025
NM_006019.4(TCIRG1):c.455_457delinsGG (p.Thr152fs)Likely pathogenic
Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2025β†’ Residue 152
NM_006019.4(TCIRG1):c.1128del (p.Asp376fs)Likely pathogenic
Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2025β†’ Residue 376
NM_006019.4(TCIRG1):c.2236C>T (p.Gln746Ter)Pathogenic
Autosomal recessive osteopetrosis 1|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 746
NM_006019.4(TCIRG1):c.504-6C>APathogenic
Osteopetrosis|not provided|Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2025
NM_006019.4(TCIRG1):c.2066G>A (p.Trp689Ter)Pathogenic
not provided|Autosomal recessive osteopetrosis 1|Osteopetrosis
β˜…β˜…β˜†β˜†2025β†’ Residue 689
NM_006019.4(TCIRG1):c.1682delinsTT (p.Gly561fs)Pathogenic
not provided|Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2025β†’ Residue 561
NM_006019.4(TCIRG1):c.2146C>T (p.Gln716Ter)Pathogenic
not provided|Autosomal recessive osteopetrosis 1
β˜…β˜…β˜†β˜†2025β†’ Residue 716
NM_006019.4(TCIRG1):c.1305+2T>CPathogenic
Autosomal recessive osteopetrosis 1|not provided
β˜…β˜…β˜†β˜†2025
View on ClinVar β†—
Related Genes
ATP6V1E2Protein interaction100%ATP6V1C2Protein interaction100%ATP6V0A2Protein interaction100%ATP12AProtein interaction99%ATP4AProtein interaction99%ATP5MC1Protein interaction99%
Tissue Expression6 tissues
Lung
100%
Liver
39%
Bone Marrow
36%
Ovary
33%
Heart
5%
Brain
1%
Gene Interaction Network
Click a node to explore
TCIRG1ATP6V1E2ATP6V1C2ATP6V0A2ATP12AATP4AATP5MC1
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q13488
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
1.22LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF1.02 [0.85–1.22]
RankingsWhere TCIRG1 stands among ~20K protein-coding genes
  • #5,005of 20,598
    Most Researched96 Β· top quartile
  • #224of 5,498
    Most Pathogenic Variants278 Β· top 5%
  • #12,862of 17,882
    Most Constrained (LOEUF)1.22
Genes detectedTCIRG1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
The different roles of V-ATPase a subunits in phagocytosis/endocytosis and autophagy.
PMID: 38873931
Autophagy Β· 2024
1.00
2
TCIRG1 and SNX10 gene mutations in the patients with autosomal recessive osteopetrosis.
PMID: 30898715
Gene Β· 2019
0.90
3
Novel Disease-Associated Missense Single-Nucleotide Polymorphisms Variants Predication by Algorithms Tools and Molecular Dynamics Simulation of Human TCIRG1 Gene Causing Congenital Neutropenia and Osteopetrosis.
PMID: 35573728
Front Mol Biosci Β· 2022
0.80
4
Malignant Infantile osteopetrosis.
PMID: 31859718
Rev Chil Pediatr Β· 2019
0.70
5
Autosomal recessive osteopetrosis: report of 41 novel mutations in the TCIRG1 gene and diagnostic implications.
PMID: 22231430
Osteoporos Int Β· 2012
0.60