ATP6V1A

Chr 3ARAD

ATPase H+ transporting V1 subunit A

Also known as: ARCL2D, ATP6A1, ATP6V1A1, DEE93, HO68, IECEE3, VA68, VPP2

This gene encodes a catalytic subunit of vacuolar H+-ATPase (V-ATPase), which acidifies intracellular organelles and maintains cellular pH homeostasis essential for protein sorting, endocytosis, and synaptic vesicle function. Mutations cause autosomal recessive cutis laxa type IID and developmental and epileptic encephalopathy 93, with both autosomal recessive and dominant inheritance patterns reported. The pathogenic mechanism involves disruption of organellar acidification, which impairs intracellular iron homeostasis and may affect neurite development and synaptic connectivity.

OMIMResearchSummary from RefSeq, OMIM, UniProt
GOFmechanismAR/ADLOEUF 0.362 OMIM phenotypes
Clinical SummaryATP6V1A
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Gene-Disease Validity (ClinGen)
autosomal recessive cutis laxa type 2D · ARLimited

Limited evidence — not for standalone diagnostic reporting

Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.87) — some intolerance to loss-of-function variants.
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ClinVar Variants
56 unique Pathogenic / Likely Pathogenic· 224 VUS of 489 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Missense constrained — critical functional residues
LoF Constraint
0.36LOEUF
pLI 0.868
Z-score 4.36
OE 0.18 (0.100.36)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
3.37Z-score
OE missense 0.49 (0.440.56)
174 obs / 352.1 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.18 (0.100.36)
00.351.4
Missense OE0.49 (0.440.56)
00.61.4
Synonymous OE0.97
01.21.6
LoF obs/exp: 6 / 33.0Missense obs/exp: 174 / 352.1Syn Z: 0.21
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongATP6V1A-related cutis laxaOTHERAR
definitiveATP6V1A-related epileptic encephalopathy, infantile or early childhoodOTHERAD
DN
0.4685th %ile
GOF
0.3788th %ile
LOF
0.59top 25%

The Badonyi & Marsh model scores loss-of-function highest, but genomic evidence most strongly supports gain-of-function as the primary mechanism.

GOF1 literature citation

Literature Evidence

GOFThese data demonstrate gain of function for p.Asp349Asn characterized by an increased proton pumping in intracellular organelles, and loss of function for p.Asp100Tyr with decreased expression of ATP6V1A and reduced levels of lysosomal markers.PMID:29668857

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

489 submitted variants in ClinVar

Classification Summary

Pathogenic42
Likely Pathogenic14
VUS224
Likely Benign163
Benign22
Conflicting4
42
Pathogenic
14
Likely Pathogenic
224
VUS
163
Likely Benign
22
Benign
4
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
13
29
0
42
Likely Pathogenic
0
13
1
0
14
VUS
16
193
11
4
224
Likely Benign
0
1
70
92
163
Benign
0
1
18
3
22
Conflicting
4
Total1622112999469

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

ATP6V1A · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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Clinical Literature
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