ATP7A

Chr XXLR

ATPase copper transporting alpha

Also known as: DSMAX, HMNX, MK, MNK, SMAX3

ATP7A encodes an ATP-driven copper pump that transfers copper ions across membranes, supplying copper to enzymes in the secretory pathway at the trans-Golgi network and exporting excess copper at the plasma membrane. Mutations cause X-linked recessive disorders including Menkes disease, occipital horn syndrome, and X-linked distal hereditary motor neuronopathy. The pathogenesis results from impaired copper homeostasis leading to deficiency of copper-dependent enzymes essential for connective tissue formation, neurotransmission, and other cellular processes.

OMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismXLRLOEUF 0.223 OMIM phenotypes
Clinical SummaryATP7A
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Gene-Disease Validity (ClinGen)
X-linked distal spinal muscular atrophy type 3 · XLModerate

Moderate evidence — consider for supplementary testing

2 total gene-disease associations curated

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.22LOEUF
pLI 1.000
Z-score 5.47
OE 0.09 (0.040.22)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
1.53Z-score
OE missense 0.82 (0.760.88)
448 obs / 548.9 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.09 (0.040.22)
00.351.4
Missense OE0.82 (0.760.88)
00.61.4
Synonymous OE0.82
01.21.6
LoF obs/exp: 4 / 42.4Missense obs/exp: 448 / 548.9Syn Z: 2.00

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

ATP7A · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗