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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 15 days ago
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ATP8A2
ATPase phospholipid transporting 8A2
Chromosome 13 Β· 13q12.13
NCBI Gene: 51761Ensembl: ENSG00000132932.19HGNC: HGNC:13533UniProt: A0A804HI09
36PubMed Papers
21Diseases
0Drugs
49Pathogenic Variants
FUNCTIONAL ROLE
Transporter
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
plasma membraneprotein bindingGolgi apparatusphosphatidylserine flippase activityDysequilibrium syndromeRare hereditary ataxiacerebellar ataxia, intellectual disability, and dysequilibriumgenetic disorder
✦AI Summary

ATP8A2 is a P4-ATPase flippase that catalyzes ATP-dependent transport of aminophospholipids, particularly phosphatidylserine (PS) and phosphatidylethanolamine (PE), from the outer to inner leaflet of cell membranes 1. As the catalytic component of the ATP8A2:TMEM30A complex, it maintains asymmetric phospholipid distribution essential for neuronal and sensory function 2. ATP8A2 is required for normal visual and auditory function, supporting photoreceptor disk membrane organization and inner ear spiral ganglion cell survival, with proposed roles in neurite outgrowth and vesicle trafficking 3. Biallelic ATP8A2 mutations cause cerebellar ataxia, impaired intellectual development, and dysequilibrium syndrome 4 (CAMRQ4), an autosomal recessive neurodevelopmental disorder 4. Clinical manifestations include severe psychomotor impairment, intellectual disability, hypotonia or spasticity, cerebellar atrophy, chorea, optic atrophy, and dental abnormalities 56. Disease-causing variants include missense mutations in catalytic domains (G447R, A772P) causing protein misfolding and degradation, and splicing variants disrupting proper mRNA processing 27. Recent evidence demonstrates ATP8A2 dysfunction leads to PS externalization and immune-mediated neurodegeneration, implicating neuroinflammatory mechanisms in CAMRQ4 pathogenesis and potentially ALS-FTD 3.

Sources cited
1
ATP8A2 translocates phosphatidylserine but not phosphatidylcholine
PMID: 34403372
2
Functional characterization of ATP8A2 variants showing misfolding and reduced ATPase activity in CAMRQ4
PMID: 38436085
3
In-frame deletion variant in ATP8A2 causes CAMRQ4 with cerebellar ataxia and intellectual disability
PMID: 39931767
4
ATP8A2 mutations associated with encephalopathy, intellectual disability, hypotonia, chorea, and optic atrophy
PMID: 27679995
5
ATP8A2 variants in catalytic domains cause protein misfolding, loss of ATPase activity, and dental abnormalities
PMID: 33565221
6
Novel ATP8A2 splicing variant identified in CAMRQ4 patient with severe psychomotor impairment
PMID: 40312603
7
ATP8A2 loss leads to phosphatidylserine externalization and immune-mediated neurodegeneration in CAMRQ4 and ALS-FTD
PMID: 41394670
Disease Associationsβ“˜21
Dysequilibrium syndromeOpen Targets
0.80Strong
Rare hereditary ataxiaOpen Targets
0.68Moderate
cerebellar ataxia, intellectual disability, and dysequilibriumOpen Targets
0.46Moderate
genetic disorderOpen Targets
0.42Moderate
hair colorOpen Targets
0.39Weak
heart failureOpen Targets
0.30Weak
COVID-19Open Targets
0.28Weak
diabetic ketoacidosisOpen Targets
0.28Weak
chronic intestinal vascular insufficiencyOpen Targets
0.27Weak
pernicious anemiaOpen Targets
0.26Weak
musculoskeletal system diseaseOpen Targets
0.26Weak
pyogenic granulomaOpen Targets
0.26Weak
ulcerative colitisOpen Targets
0.25Weak
subarachnoid hemorrhageOpen Targets
0.24Weak
idiopathic pulmonary fibrosisOpen Targets
0.22Weak
severe acute respiratory syndromeOpen Targets
0.22Weak
epilepsyOpen Targets
0.11Weak
Severe intellectual disabilityOpen Targets
0.11Weak
spinocerebellar ataxia type 4Open Targets
0.08Suggestive
MODYOpen Targets
0.08Suggestive
Cerebellar ataxia, impaired intellectual development, and dysequilibrium syndrome 4UniProt
Pathogenic Variants49
NM_016529.6(ATP8A2):c.77-2A>GPathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4|not provided
β˜…β˜…β˜†β˜†2025
NM_016529.6(ATP8A2):c.3272+1G>APathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4|not provided
β˜…β˜…β˜†β˜†2025
NM_016529.6(ATP8A2):c.1782+2T>CPathogenic
not provided|Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜…β˜†β˜†2025
NM_016529.6(ATP8A2):c.1761dup (p.Arg588fs)Pathogenic
Inborn genetic diseases|Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4|not provided|ATP8A2-related disorder
β˜…β˜…β˜†β˜†2024β†’ Residue 588
NM_016529.6(ATP8A2):c.2293G>T (p.Asp765Tyr)Likely pathogenic
not provided|Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜…β˜†β˜†2024β†’ Residue 765
NM_016529.6(ATP8A2):c.2212-1G>CPathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜…β˜†β˜†2024
NM_016529.6(ATP8A2):c.1756C>T (p.Arg586Ter)Pathogenic
not provided|Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜…β˜†β˜†2022β†’ Residue 586
NM_016529.6(ATP8A2):c.2733G>A (p.Trp911Ter)Likely pathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜†β˜†β˜†2026β†’ Residue 911
NM_016529.6(ATP8A2):c.812T>G (p.Leu271Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 271
NM_016529.6(ATP8A2):c.3076-2A>GLikely pathogenic
not provided
β˜…β˜†β˜†β˜†2024
NM_016529.6(ATP8A2):c.3296_3297delinsGCAAGCACAC (p.Thr1099fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 1099
NM_016529.6(ATP8A2):c.1484del (p.Pro495fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 495
NM_016529.6(ATP8A2):c.649C>T (p.Gln217Ter)Pathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜†β˜†β˜†2024β†’ Residue 217
NM_016529.6(ATP8A2):c.3316dup (p.Glu1106fs)Pathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜†β˜†β˜†2024β†’ Residue 1106
NM_016529.6(ATP8A2):c.1287G>T (p.Lys429Asn)Likely pathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜†β˜†β˜†2024β†’ Residue 429
NM_016529.6(ATP8A2):c.3334C>T (p.Arg1112Ter)Likely pathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜†β˜†β˜†2024β†’ Residue 1112
NM_016529.6(ATP8A2):c.1741C>T (p.Arg581Ter)Pathogenic
Cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 4
β˜…β˜†β˜†β˜†2024β†’ Residue 581
NM_016529.6(ATP8A2):c.1110C>G (p.Tyr370Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2023β†’ Residue 370
NM_016529.6(ATP8A2):c.3361dup (p.Asp1121fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2023β†’ Residue 1121
NM_016529.6(ATP8A2):c.1170_1174delinsAAGTATACTCAAGTATACTCAAGTATACTCAAGTATACTCAAGTATAC (p.Leu391fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2023β†’ Residue 391
View on ClinVar β†—
Related Genes
CDC50AProtein interaction94%RNF6Protein interaction85%ATP11CShared pathway27%ATP11AShared pathway25%ATP8A1Shared pathway23%ATP8B3Shared pathway18%
Tissue Expression6 tissues
Brain
100%
Bone Marrow
2%
Lung
2%
Ovary
1%
Heart
1%
Liver
0%
Gene Interaction Network
Click a node to explore
ATP8A2CDC50ARNF6ATP11CATP11AATP8A1ATP8B3
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q6ZU25
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.73LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.58 [0.46–0.73]
RankingsWhere ATP8A2 stands among ~20K protein-coding genes
  • #10,737of 20,598
    Most Researched36
  • #1,361of 5,498
    Most Pathogenic Variants49 Β· top quartile
  • #5,665of 17,882
    Most Constrained (LOEUF)0.73
Genes detectedATP8A2
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Chorea in children: etiology, diagnostic approach and management.
PMID: 32776155
J Neural Transm (Vienna) Β· 2020
1.00
2
Autozygome and high throughput confirmation of disease genes candidacy.
PMID: 30237576
Genet Med Β· 2019
0.90
3
New ATP8A2 gene mutations associated with a novel syndrome: encephalopathy, intellectual disability, severe hypotonia, chorea and optic atrophy.
PMID: 27679995
Neurogenetics Β· 2016
0.80
4
Reevaluation of the Impact of the Novel Likely Pathogenic Variant c.1286_1288delAGA in the ATP8A2 Gene: A 7-Year Follow-Up With Clinical, Genetic, and ACMG Insights in an Iranian Family.
PMID: 39931767
Mol Genet Genomic Med Β· 2025
0.70
5
Functional and in silico analysis of ATP8A2 and other P4-ATPase variants associated with human genetic diseases.
PMID: 38436085
Dis Model Mech Β· 2024
0.60