ATP6V0A4

Chr 7AR

ATPase H+ transporting V0 subunit a4

Also known as: A4, ATP6N1B, ATP6N2, DRTA3, RDRTA2, RTA1C, RTADR, STV1

This gene encodes a component of vacuolar ATPase (V-ATPase), a multisubunit enzyme that mediates acidification of intracellular compartments of eukaryotic cells. V-ATPase dependent acidification is necessary for such intracellular processes as protein sorting, zymogen activation, receptor-mediated endocytosis, and synaptic vesicle proton gradient generation. V-ATPase is composed of a cytosolic V1 domain and a transmembrane V0 domain. The V1 domain consists of three A and three B subunits, two G subunits plus the C, D, E, F, and H subunits. The V1 domain contains the ATP catalytic site. The V0 domain consists of five different subunits: a, c, c', c'', and d. This gene is one of four genes in man and mouse that encode different isoforms of the a subunit. Alternatively spliced transcript variants encoding the same protein have been described. Mutations in this gene are associated with renal tubular acidosis associated with preserved hearing. [provided by RefSeq, Jul 2008]

GeneReviewsOMIMResearchGenerating clinical summary…
MultiplemechanismARLOEUF 1.001 OMIM phenotype
Clinical SummaryATP6V0A4
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
88 unique Pathogenic / Likely Pathogenic· 269 VUS of 655 total submissions
📖
GeneReview available — ATP6V0A4
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint?
1.00LOEUF
pLI 0.000
Z-score 1.53
OE 0.76 (0.591.00)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint?
0.85Z-score
OE missense 0.89 (0.820.96)
408 obs / 459.6 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios?
LoF OE?0.76 (0.591.00)
00.351.4
Missense OE?0.89 (0.820.96)
00.61.4
Synonymous OE?0.99
01.21.6
LoF obs/exp: 37 / 48.5Missense obs/exp: 408 / 459.6Syn Z: 0.08

This gene — mechanism propensity

DN
0.6839th %ile
GOF
0.7127th %ile
LOF
0.2873th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function and dominant-negative). Both the Badonyi & Marsh prediction and the broader genomic evidence point to gain-of-function as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

GOFprediction above median
DNprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

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

ClinVar Variant Classifications

655 submitted variants in ClinVar

Classification Summary

Pathogenic32
Likely Pathogenic56
VUS269
Likely Benign147
Benign110
Conflicting23
32
Pathogenic
56
Likely Pathogenic
269
VUS
147
Likely Benign
110
Benign
23
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
30
1
0
1
32
Likely Pathogenic
44
12
0
0
56
VUS
1
227
31
10
269
Likely Benign
0
10
74
63
147
Benign
1
4
96
9
110
Conflicting
23
Total7625420183637

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

View in ClinVar →

44 pathogenic / likely-pathogenic (of 52) ClinVar copy-number / structural variants overlap ATP6V0A4 — these span large chromosomal regions, not the gene specifically, and are excluded from the counts above. Explore in CNV tools →

Protein Context — Lollipop Plot

ATP6V0A4 · protein map & ClinVar variants

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

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →