HomeAboutRankingsData Sources
© 2026 GeneE
🧬
GeneE
10 sources retrieved · Most recent: April 2026 · Index updated 15 days ago
ⓘGeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
OSTM1
osteoclastogenesis associated transmembrane protein 1
Chromosome 6 · 6q21
NCBI Gene: 28962Ensembl: ENSG00000081087.16HGNC: HGNC:21652UniProt: A0A8V8TNX7
46PubMed Papers
21Diseases
0Drugs
27Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
protein bindingtransepithelial chloride transportlysosomal membraneosteoclast differentiationinfantile osteopetrosis with neuroaxonal dysplasiaAutosomal recessive malignant osteopetrosisosteopetrosispsoriasis
✦AI Summary

OSTM1 (osteoclastogenesis associated transmembrane protein 1) is a type I transmembrane protein essential for osteoclast and melanocyte maturation and function 1. As the β-subunit of the ClC-7 chloride/proton exchanger, OSTM1 facilitates 2Cl−/H+ exchange across lysosomal membranes, critical for maintaining lysosomal pH gradients and bone resorption 2. Mechanistically, OSTM1 functions bifunctionally in osteoclasts: it negatively regulates preosteoclast fusion by controlling intracellular calcium signaling and Nfatc1 nuclear translocation, while also regulating endolysosomal trafficking and exocytosis of bone-degrading enzymes like cathepsin K and tartrate-resistant acid phosphatase 3. OSTM1 localizes to the endoplasmic reticulum, trans-Golgi network, and endosomes/lysosomes, where it forms a cytosolic scaffolding complex with kinesin 5B for intracellular cargo trafficking 4. Mutations in OSTM1 cause autosomal recessive osteopetrosis type 5 (OPTB5), the most severe form of osteopetrosis, characterized by abnormally increased bone density, bone marrow occlusion, hematopoietic defects, and neurodegeneration leading to early death 5. Loss-of-function mutations impair osteoclast bone-resorbing capacity, whereas gain-of-function CLCN7 variants cause distinct lysosomal pathology without osteopetrosis 2. Recent studies identify OSTM1 as an E3 ubiquitin ligase involved in copper metabolism regulation 6.

Sources cited
1
OSTM1 is essential for osteoclast maturation and responsible for severe osteopetrosis when mutated
PMID: 32764302
2
OSTM1 is the β-subunit of ClC-7 performing 2Cl−/H+ exchange across lysosomal membranes; loss-of-function causes osteopetrosis and lysosomal storage
PMID: 38838776
3
OSTM1 negatively regulates preosteoclast fusion and controls endolysosomal trafficking and exocytosis of bone-degrading enzymes
PMID: 29297601
4
OSTM1 forms a cytosolic scaffolding complex with kinesin 5B and localizes to ER, trans-Golgi network, and endosomes/lysosomes for intracellular trafficking
PMID: 26598607
5
OSTM1 mutations cause autosomal recessive osteopetrosis type 5 (OPTB5), the most severe form, with onset in infancy and early mortality
PMID: 35902071
6
OSTM1 functions as an E3 ligase that ubiquitinates ATP7A to regulate copper metabolism
PMID: 40838985
Disease Associationsⓘ21
infantile osteopetrosis with neuroaxonal dysplasiaOpen Targets
0.74Strong
Autosomal recessive malignant osteopetrosisOpen Targets
0.66Moderate
osteopetrosisOpen Targets
0.45Moderate
psoriasisOpen Targets
0.33Weak
alopecia areataOpen Targets
0.23Weak
genetic disorderOpen Targets
0.19Weak
neoplasmOpen Targets
0.10Suggestive
colorectal carcinomaOpen Targets
0.08Suggestive
gastric cancerOpen Targets
0.08Suggestive
posterior cortical atrophyOpen Targets
0.08Suggestive
infectionOpen Targets
0.08Suggestive
renal cell carcinomaOpen Targets
0.08Suggestive
cancerOpen Targets
0.07Suggestive
tooth agenesisOpen Targets
0.07Suggestive
tricho-dento-osseous syndromeOpen Targets
0.07Suggestive
OligodontiaOpen Targets
0.06Suggestive
Griscelli diseaseOpen Targets
0.06Suggestive
amelogenesis imperfectaOpen Targets
0.06Suggestive
esophageal squamous cell carcinomaOpen Targets
0.06Suggestive
Hypomaturation amelogenesis imperfectaOpen Targets
0.06Suggestive
Osteopetrosis, autosomal recessive 5UniProt
Pathogenic Variants27
NM_014028.4(OSTM1):c.25C>T (p.Gln9Ter)Pathogenic
not provided|Autosomal recessive osteopetrosis 5
★★☆☆2024→ Residue 9
NM_014028.4(OSTM1):c.692dup (p.Ser232fs)Pathogenic
not provided|Autosomal recessive osteopetrosis 5
★★☆☆2023→ Residue 232
NM_014028.4(OSTM1):c.415_416del (p.Gln140fs)Pathogenic
Autosomal recessive osteopetrosis 5|not provided
★★☆☆2023→ Residue 140
NM_014028.4(OSTM1):c.796C>T (p.Arg266Ter)Pathogenic
not provided
★☆☆☆2025→ Residue 266
NM_014028.4(OSTM1):c.498G>A (p.Trp166Ter)Pathogenic
not provided
★☆☆☆2025→ Residue 166
NM_014028.4(OSTM1):c.811C>T (p.Arg271Ter)Pathogenic
Autosomal recessive osteopetrosis 5
★☆☆☆2024→ Residue 271
NM_014028.4(OSTM1):c.551del (p.Asn184fs)Pathogenic
not provided
★☆☆☆2024→ Residue 184
NM_014028.4(OSTM1):c.74G>A (p.Trp25Ter)Pathogenic
not provided
★☆☆☆2024→ Residue 25
NM_014028.4(OSTM1):c.51G>A (p.Trp17Ter)Pathogenic
not provided
★☆☆☆2024→ Residue 17
NM_014028.4(OSTM1):c.75G>A (p.Trp25Ter)Pathogenic
not provided
★☆☆☆2024→ Residue 25
NM_014028.4(OSTM1):c.548C>G (p.Ser183Ter)Pathogenic
not provided
★☆☆☆2024→ Residue 183
NM_014028.4(OSTM1):c.517+1G>TLikely pathogenic
not provided
★☆☆☆2024
NM_014028.4(OSTM1):c.402_402+1dupPathogenic
not provided
★☆☆☆2024
NM_014028.4(OSTM1):c.358C>T (p.Gln120Ter)Pathogenic
not provided
★☆☆☆2023→ Residue 120
NM_014028.4(OSTM1):c.421_422del (p.Ser141fs)Pathogenic
not provided
★☆☆☆2023→ Residue 141
NM_014028.4(OSTM1):c.256G>T (p.Glu86Ter)Pathogenic
not provided
★☆☆☆2023→ Residue 86
NM_014028.4(OSTM1):c.560del (p.Val187fs)Pathogenic
not provided
★☆☆☆2023→ Residue 187
NM_014028.4(OSTM1):c.255_256delinsC (p.Glu86fs)Likely pathogenic
Osteopetrosis
★☆☆☆2023→ Residue 86
NM_014028.4(OSTM1):c.402+1G>ALikely pathogenic
not provided
★☆☆☆2022
NM_014028.4(OSTM1):c.564T>G (p.Tyr188Ter)Pathogenic
not provided
★☆☆☆2022→ Residue 188
View on ClinVar ↗
Related Genes
TCIRG1Protein interaction96%PLEKHM1Protein interaction92%CLCN6Protein interaction82%CA2Protein interaction80%SNX10Protein interaction78%TNFRSF11AProtein interaction78%
Tissue Expression6 tissues
Heart
100%
Lung
70%
Brain
68%
Liver
41%
Ovary
37%
Bone Marrow
24%
Gene Interaction Network
Click a node to explore
OSTM1TCIRG1PLEKHM1CLCN6CA2SNX10TNFRSF11A
PROTEIN STRUCTURE
Preparing viewer…
PDB9G6C · 1.80 Å · EM
View on RCSB ↗
Constraintⓘ
LOEUFⓘ
1.43LoF Tolerant
pLIⓘ
0.00Tolerant
Observed/Expected LoF0.91 [0.60–1.43]
RankingsWhere OSTM1 stands among ~20K protein-coding genes
  • #9,357of 20,598
    Most Researched46
  • #1,914of 5,498
    Most Pathogenic Variants27
  • #14,679of 17,882
    Most Constrained (LOEUF)1.43
Genes detectedOSTM1
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
Targeting ATP7A-Dependent Copper Metabolic Homeostasis Induces Cuproptosis and Suppresses the Progression of Mutant KRAS-Driven Lung Cancer.
PMID: 40838985
Cancer Res · 2025
1.00
2
Gain-of-function variants in CLCN7 cause hypopigmentation and lysosomal storage disease.
PMID: 38838776
J Biol Chem · 2024
0.90
3
Ostm1 from Mouse to Human: Insights into Osteoclast Maturation.
PMID: 32764302
Int J Mol Sci · 2020
0.80
4
Role of Ostm1 Cytosolic Complex with Kinesin 5B in Intracellular Dispersion and Trafficking.
PMID: 26598607
Mol Cell Biol · 2016
0.70
5
Expression pattern of the V5-Ostm1 protein in bacterial artificial chromosome transgenic mice.
PMID: 33484096
Genesis · 2021
0.60