RARB

Chr 3ADAR

retinoic acid receptor beta

Also known as: HAP, MCOPS12, NR1B2, RARbeta, RARbeta1, RRB2

This gene encodes retinoic acid receptor beta, a nuclear transcriptional regulator that binds retinoic acid (vitamin A) and forms heterodimers to regulate gene expression during embryonic development, cell growth, and differentiation. Mutations cause syndromic microphthalmia 12 with both autosomal dominant and autosomal recessive inheritance patterns reported. The gene is highly constrained against loss-of-function variants (pLI 0.9998, LOEUF 0.12), indicating that complete loss of protein function is typically incompatible with normal development.

OMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismAD/ARLOEUF 0.121 OMIM phenotype
Clinical SummaryRARB
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Gene-Disease Validity (ClinGen)
microphthalmia, syndromic 12 · 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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ClinVar Variants
46 unique Pathogenic / Likely Pathogenic· 72 VUS of 192 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.12LOEUF
pLI 1.000
Z-score 4.61
OE 0.00 (0.000.12)
Highly constrained

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

Missense Constraint
2.87Z-score
OE missense 0.49 (0.420.57)
123 obs / 250.7 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.00 (0.000.12)
00.351.4
Missense OE0.49 (0.420.57)
00.61.4
Synonymous OE1.24
01.21.6
LoF obs/exp: 0 / 24.8Missense obs/exp: 123 / 250.7Syn Z: -1.82
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveRARB-related microphthalmia syndromicGOFAD
definitiveRARB-related microphthalmia syndromicLOFAR
DN
0.2997th %ile
GOF
0.3292th %ile
LOF
0.82top 5%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function and gain-of-function). 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 · 17% of P/LP variants are LoF · LOEUF 0.12
GOF1 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

GOFGain-of-Function Mutations in RARB Cause Intellectual Disability with Progressive Motor Impairment.PMID:27120018

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

192 submitted variants in ClinVar

Classification Summary

Pathogenic24
Likely Pathogenic22
VUS72
Likely Benign27
Benign23
Conflicting3
24
Pathogenic
22
Likely Pathogenic
72
VUS
27
Likely Benign
23
Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
3
18
0
24
Likely Pathogenic
5
16
1
0
22
VUS
12
52
8
0
72
Likely Benign
0
6
10
11
27
Benign
0
2
18
3
23
Conflicting
3
Total20795514171

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

View in ClinVar →

Protein Context — Lollipop Plot

RARB · 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

No active trials found for this gene.

Search ClinicalTrials.gov →
Clinical Literature
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