CTNNB1

Chr 3AD

catenin beta 1

Also known as: CTNNB, EVR7, MRD19, NEDSDV, armadillo

This protein functions in cell adhesion by constituting adherens junctions necessary for epithelial cell layer formation, anchoring the actin cytoskeleton, and transmitting contact inhibition signals to regulate cell division. Autosomal dominant loss-of-function mutations cause a neurodevelopmental disorder with spastic diplegia and visual defects, as well as exudative vitreoretinopathy. The protein is extremely intolerant to loss-of-function variants, with somatic mutations also associated with various cancers including medulloblastoma.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismADLOEUF 0.137 OMIM phenotypes
Clinical SummaryCTNNB1
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Gene-Disease Validity (ClinGen)
CTNNB1-related neurodevelopmental disorder and/or vitreoretinopathy · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

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
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Dual constrained — LoF & missense intolerant
LoF Constraint
0.13LOEUF
pLI 1.000
Z-score 5.46
OE 0.03 (0.010.13)
Highly constrained

Among the most LoF-intolerant genes (~top 3%)

Missense Constraint
3.85Z-score
OE missense 0.47 (0.420.53)
199 obs / 421.1 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.03 (0.010.13)
00.351.4
Missense OE0.47 (0.420.53)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 1 / 36.7Missense obs/exp: 199 / 421.1Syn Z: 0.14
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveCTNNB1-related exudative vitreoretinopathyLOFAD
definitiveCTNNB1-related intellectual developmental disorderLOFAD
DN
0.4784th %ile
GOF
0.7028th %ile
LOF
0.88top 5%

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

LOFprediction above median · 1 literature citation · LOEUF 0.13
GOFprediction above median · 1 literature citation
DN1 literature citation

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.

Literature Evidence

DNThe batface mutant expresses a unique phenotype that is a combined consequence not only of a gain in WNT-related function (15), but also, as we show here, a dominant-negative effect on adhesion-related function.PMID:24614104
GOFFinally, in a few MODY3 patients with HNF1A germline mutation leading to amino acid substitutions outside the POU-H domain, we identified a different subtype of HCA either with a gp130 and/or CTNNB1 activating mutation.PMID:20393147
LOFA new intellectual disability syndrome caused by CTNNB1 haploinsufficiency.PMID:24668549

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

CTNNB1 · 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

Colorectal Cancer

ASPirin Intervention for the REDuction of Colorectal Cancer Risk

ACTIVE NOT RECRUITING
NCT02394769Phase NAMassachusetts General HospitalStarted 2015-07-06
AspirinPlacebo for Aspirin
Lung Cancer

Feasibility of Targeted Bronchial Washing for Molecular Testing by Next Generation Sequencing in Early-stage Lung Cancer

ACTIVE NOT RECRUITING
NCT06301295Phase NAPusan National University HospitalStarted 2024-05-29
Ultarthin bronchoscopy with intratumoral washing
16P11.2 Deletion Syndrome16p11.2 Duplications1Q21.1 Deletion

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

RECRUITING
NCT01238250Simons SearchlightStarted 2010-10
Craniopharyngioma, Child

Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

ENROLLING BY INVITATION
NCT03610906University of Colorado, DenverStarted 2019-03-12
Tumor and Blood Specimens
Advanced Hepatocellular CarcinomaMetastatic Hepatocellular CarcinomaStage III Hepatocellular Carcinoma AJCC v8

Testing the Addition of an Anti-cancer Drug, Sapanisertib, to the Usual Chemotherapy Treatment (Cabozantinib) in Metastatic Liver Cell Cancer With a Change in Genes for the Protein β-Catenin, The SAPHIRE Trial

RECRUITING
NCT06811116Phase PHASE1, PHASE2National Cancer Institute (NCI)Started 2025-11-17
Biospecimen CollectionCabozantinib S-malateImaging Procedure
CTNNB1 Neurodevelopmental Syndrome

CTNNB1 Neurodevelopmental Syndrome - Natural History Study

RECRUITING
NCT07167732University Medical Centre LjubljanaStarted 2024-06-14
A General Medical and Neurological AssessmentWorld Health Organisation (WHO) Motor MilestonesBurke-Fahn-Marsden Dystonia Rating Scale
Gastric CancerHealthy

Preliminary Experimental Study on Key Technologies for Early Screening of Gastric Cancer

RECRUITING
NCT05991947Zhejiang Cancer HospitalStarted 2021-03-01
No intervention
Post Traumatic Stress DisorderComplex Posttraumatic Stress DisorderStress

Building Resilience at Schools: Emotional and Biological Assessment and Treatment of Traumatic Stress

RECRUITING
NCT05701111Phase NAStanford UniversityStarted 2024-02-23
Start with the Heart StudentsStart with the Heart TeachersCue Centered Therapy Counselors
Unresectable Colorectal Neoplasm Metastasis

Study of a Tankyrase Inhibitor RK-582 for Patients With Unresectable Metastatic Colorectal Cancer

RECRUITING
NCT06853496Phase PHASE1Eiji ShinozakiStarted 2025-03-13
RK-582
Metastatic Colorectal Carcinoma (mCRC)Colorectal Cancer (CRC)Adenomatous Polyposis Coli (APC) Gene Mutation

Safety and Efficacy of Tegavivint in Patients With Metastatic Colorectal Carcinoma

RECRUITING
NCT07463599Phase PHASE1, PHASE2HonorHealth Research InstituteStarted 2026-02-17
Tegavivint
Desmoid FibromatosisRecurrent Desmoid FibromatosisUnresectable Desmoid Fibromatosis

A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery

ACTIVE NOT RECRUITING
NCT04195399Phase PHASE2Children's Oncology GroupStarted 2020-10-07
Biospecimen CollectionComputed TomographyEchocardiography Test
Advanced Hepatocellular CarcinomaMetastatic Hepatocellular Carcinoma

A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma

RECRUITING
NCT06600321Phase PHASE1Alnylam PharmaceuticalsStarted 2024-12-30
ALN-BCATPembrolizumab
Clinical Literature
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Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
Top 5 full-text resultsSearch PubTator3 ↗