ATP7B

Chr 13AR

ATPase copper transporting beta

Also known as: PWD, WC1, WD, WND

This gene is a member of the P-type cation transport ATPase family and encodes a protein with several membrane-spanning domains, an ATPase consensus sequence, a hinge domain, a phosphorylation site, and at least 2 putative copper-binding sites. This protein is a monomer, and functions as a copper-transporting ATPase which exports copper out of the cells, such as the efflux of hepatic copper into the bile. Alternate transcriptional splice variants, encoding different isoforms with distinct cellular localizations, have been characterized. Mutations in this gene have been associated with Wilson disease which is characterized by copper accumulation. [provided by RefSeq, Dec 2019]

GeneReviewsOMIMResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

Wilson diseaseMIM #277900
AR

Clinical highlights

Gene-disease validity (ClinGen)
Wilson disease · ARDefinitivesufficient evidence for diagnostic panels
11
Active trials
260
Pubs (1 yr)
P/LP submissions
P/LP missense
1.19
LOEUF
Multiple*
Mechanism· predicted
📖
GeneReview available — ATP7B
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?
1.19LOEUF
pLI 0.000
Z-score 0.45
OE 0.93 (0.741.19)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint?
-0.88Z-score
OE missense 1.09 (1.031.15)
885 obs / 814.3 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios?
LoF OE?0.93 (0.741.19)
00.351.4
Missense OE?1.09 (1.031.15)
00.61.4
Synonymous OE?1.07
01.21.6
LoF obs/exp: 47 / 50.4Missense obs/exp: 885 / 814.3Syn Z: -1.02

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

ATP7B · 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

Wilson DiseaseD-PenicillamineEffect of D-penicilline on Cutaneous Elastity of Wilson's Patient

Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity

RECRUITING
NCT06945081Phase NACentre Hospitalier Universitaire de Saint EtienneStarted 2025-11-28
Preparation of forearm molds with SILFLO® silicone
Wilson DiseaseWilson's DiseaseWilsons Disease

A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

RECRUITING
NCT07748403Phase PHASE1, PHASE2Prime Medicine, Inc.Started 2026-09
PM577a
Wilson Disease

A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease

RECRUITING
NCT06650319Phase EARLY_PHASE1Chaohui YuStarted 2024-09-24
LY-M003
Wilson's Disease

Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's Disease

RECRUITING
NCT05183165Phase NAFondation Ophtalmologique Adolphe de RothschildStarted 2022-05-11
Patients with Wilson's disease declaring pregnancy,
Wilson's Disease

Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map

NOT YET RECRUITING
NCT07241832Assiut UniversityStarted 2025-12-01
KAB-pB Scores
Wilson Disease

An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease

ACTIVE NOT RECRUITING
NCT06663878Phase NAXinhua Hospital, Shanghai Jiao Tong University School of MedicineStarted 2024-10-28
MWAV201
Wilson Disease

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

ACTIVE NOT RECRUITING
NCT04884815Phase PHASE1, PHASE2Ultragenyx Pharmaceutical IncStarted 2021-09-27
UX701Standard of Care (SOC)
Wilson Disease

Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)

NOT YET RECRUITING
NCT07173933Phase PHASE1, PHASE2GeneCradle IncStarted 2025-10
GC310
Wilson's Disease

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

ACTIVE NOT RECRUITING
NCT04537377Phase PHASE1, PHASE2Vivet Therapeutics SASStarted 2021-09-03
VTX-801
Wilson Disease

Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT

ENROLLING BY INVITATION
NCT07159581Thomas Damgaard SandahlStarted 2025-07-08
Wilson Disease

Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

ACTIVE NOT RECRUITING
NCT07226622Prime Medicine, Inc.Started 2025-12-29