RAP1B

Chr 12AD

RAP1B, member of RAS oncogene family

Also known as: K-REV, RAL1B, THC11

RAP1B encodes a small GTP-binding protein that regulates integrin-mediated cell signaling, platelet function, and endothelial cell polarity and barrier function. Mutations cause thrombocytopenia 11 with multiple congenital anomalies and dysmorphic facies, inherited in an autosomal dominant pattern. The gene is highly constrained against loss-of-function variants (pLI 0.91, LOEUF 0.39), indicating that functional copies are essential for normal development.

Summary from RefSeq, OMIM, UniProt
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Primary Disease Associations & Inheritance

Thrombocytopenia 11 with multiple congenital anomalies and dysmorphic faciesMIM #620654
AD
0
Active trials
37
Pubs (1 yr)
19
P/LP submissions
35%
P/LP missense
0.39
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryRAP1B
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Gene-Disease Validity (ClinGen)
thrombocytopenia 11 with multiple congenital anomalies and dysmorphic facies · ADModerate

Moderate evidence — consider for supplementary testing

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 0.91). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
17 unique Pathogenic / Likely Pathogenic· 12 VUS of 69 total submissions
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GeneReview available — RAP1B
Authoritative clinical overview · Recommended first read
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.39LOEUF
pLI 0.910
Z-score 2.96
OE 0.08 (0.030.39)
Highly constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
2.60Z-score
OE missense 0.25 (0.180.35)
24 obs / 95.5 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.08 (0.030.39)
00.351.4
Missense OE0.25 (0.180.35)
00.61.4
Synonymous OE1.10
01.21.6
LoF obs/exp: 1 / 12.1Missense obs/exp: 24 / 95.5Syn Z: -0.45
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedRAP1B-related developmental disorderOTHERAD
DN
0.6065th %ile
GOF
0.7030th %ile
LOF
0.52top 25%

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

GOFprediction above median · 1 literature citation
LOFLOEUF 0.39

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

GOFWe report a 23-year-old male with a novel, de novo RAP1B gain-of-function variant identified on genome sequencing.PMID:35451551

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

69 submitted variants in ClinVar

Classification Summary

Pathogenic14
Likely Pathogenic3
VUS12
Likely Benign7
Benign14
Conflicting1
14
Pathogenic
3
Likely Pathogenic
12
VUS
7
Likely Benign
14
Benign
1
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
4
10
0
14
Likely Pathogenic
0
2
1
0
3
VUS
1
9
2
0
12
Likely Benign
0
0
2
5
7
Benign
0
0
13
1
14
Conflicting
1
Total11528651

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

View in ClinVar →

Protein Context — Lollipop Plot

RAP1B · 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

No active trials found for this gene.

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Clinical Literature
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