CAD

Chr 2AR

carbamoyl-phosphate synthetase 2, aspartate transcarbamylase, and dihydroorotase

Also known as: CDG1Z, DEE50, EIEE50, GATD4

The de novo synthesis of pyrimidine nucleotides is required for mammalian cells to proliferate. This gene encodes a trifunctional protein which is associated with the enzymatic activities of the first 3 enzymes in the 6-step pathway of pyrimidine biosynthesis: carbamoylphosphate synthetase (CPS II), aspartate transcarbamoylase, and dihydroorotase. This protein is regulated by the mitogen-activated protein kinase (MAPK) cascade, which indicates a direct link between activation of the MAPK cascade and de novo biosynthesis of pyrimidine nucleotides. Alternative splicing results in multiple transcript variants encoding different isoforms. [provided by RefSeq, Apr 2015]

OMIMResearchGenerating clinical summary…
LOFmechanismARLOEUF 0.251 OMIM phenotype
Clinical SummaryCAD
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Gene-Disease Validity (ClinGen)
developmental and epileptic encephalopathy, 50 · ARDefinitive

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?
0.25LOEUF
pLI 1.000
Z-score 8.09
OE 0.17 (0.120.25)
Highly constrained

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

Missense Constraint?
4.34Z-score
OE missense 0.67 (0.630.71)
913 obs / 1363.6 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios?
LoF OE?0.17 (0.120.25)
00.351.4
Missense OE?0.67 (0.630.71)
00.61.4
Synonymous OE?1.05
01.21.6
LoF obs/exp: 19 / 110.9Missense obs/exp: 913 / 1363.6Syn Z: -0.93
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveCAD-related uridine-responsive epileptic encephalopathyLOFAR

This gene — mechanism propensity

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.4289th %ile
GOF
0.3986th %ile
LOF
0.64top 25%

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

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

CAD · 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

Coronary Artery Disease

Safety and Efficacy of Meplazumab in Patients With Coronary Artery Disease

RECRUITING
NCT06572267Phase PHASE2Xijing HospitalStarted 2024-10-16
Mepolizumab low dose groupMepolizumab middle dose groupMepolizumab high dose group
Familial Hypercholesterolemia

EAS Familial Hypercholesterolaemia Studies Collaboration

RECRUITING
NCT04272697Imperial College LondonStarted 2015-03-22
Acute Myeloid Leukemia

Combination of Midostaurin and Gemtuzumab Ozogamicin in First-line Standard Therapy for Acute Myeloid Leukemia (MOSAIC)

RECRUITING
NCT04385290Phase PHASE1, PHASE2Technische Universität DresdenStarted 2020-09-04
MODULE: conventional chemotherapy (Cytarabine+Daunorubicin) in combination with midostaurin+GOMAGNOLIA-trial: Midostaurin associated with conventional chemotherapy (AraC+DNR)+GOMAGNOLIA-trial: conventional chemotherapy (AraC+DNR)+GO
PRS-based Primary Prevention of CVD

Polygenic Risk Driven Pragmatic Statin Trial for Heart Disease Prevention

ACTIVE NOT RECRUITING
NCT06820086Phase PHASE4Mikk JÜRISSONStarted 2025-03-26
Rosuvastatin 20mg
Coronary Arterial Disease (CAD)

Health Improvements by Understanding the Determinants of Residual Risk in Coronary Artery Disease and New Targets for Prevention and Treatment

RECRUITING
NCT06601153Fondazione Toscana Gabriele MonasterioStarted 2023-07-07
Cardiac CT
Coronary Artery DiseaseArterial Stiffness, Blood PressureAORTIC VALVE DISEASES

Expanded Studies on the SCAPIS Stockholm Reexamination Cohort

RECRUITING
NCT07476703Danderyd HospitalStarted 2024-04-08
Coronary computer tomography (CCTA)Echocardiograhy and aortic ultrasoundLiver elastography
Ischemic Heart DiseaseChronic Coronary SyndromeOptical Coherence Tomography (OCT)

Evaluating the Role of IGF-1 and S-Klotho In Plaque Phenotype and Vulnerability: the VISION Study.

ACTIVE NOT RECRUITING
NCT06522074Fondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2024-10-15
Coronary angiography with OCT
Coronary Artery Disease

A Precision Medicine Approach to Identify Patients Undergoing Elective PCI at Risk of Peri-PCI Myocardial Infarction

RECRUITING
NCT05332262University of FloridaStarted 2022-05-23
Assessment of platelet reactivityTroponin measurement
Coronary Arterial Disease (CAD)Percutaneous Coronary Intervention (PCI)

Dual Antiplatelet Therapy Escalation From Standard-dose Clopidogrel to Low-Dose Prasugrel in Patients With High Bleeding and Ischemic Risk Undergoing PCI: A Prospective, Randomized Pharmacodynamic Study (TAILOR-BLEED-2)

RECRUITING
NCT07025148Phase PHASE4University of FloridaStarted 2025-10-01
PrasugrelClopidogrel
Lung Cancer

SingapOre Lung Cancer Screening Through Integrating CT With Other biomarkErs (SOLSTICE)

RECRUITING
NCT05724264Phase NANational Cancer Centre, SingaporeStarted 2022-07-21
Low-dose CT of the Chest (LDCT) + Sampling for Biomarker Assays
Healthy

The Healthy Elderly Longevity Cohort

RECRUITING
NCT01004133Scripps Translational Science InstituteStarted 2007-08
Periodontal DiseasesAcute Coronary Artery Disease

Klotho Gene, Red Complex Bacteria and Periodontal Viruses in Patients With and Without Periodontitis and Acute Coronary Syndrome

NOT YET RECRUITING
NCT07504744Meenakshi Ammal Dental College and HospitalStarted 2026-03-31