RBM20

Chr 10AD

RNA binding motif protein 20

This highly constrained gene encodes an RNA-binding protein that regulates alternative splicing of cardiac structural proteins including titin, specifically binding intronic sequences to promote exon skipping during cardiac development. Mutations cause autosomal dominant dilated cardiomyopathy (cardiomyopathy, dilated, 1DD), which primarily affects cardiac muscle function. The gene's high constraint against loss-of-function variants (pLI 0.99, LOEUF 0.30) suggests intolerance to protein-disrupting mutations.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
MultiplemechanismADLOEUF 0.291 OMIM phenotype
Clinical SummaryRBM20
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Gene-Disease Validity (ClinGen)
hypertrophic cardiomyopathy · ADLimited

Limited evidence — not for standalone diagnostic reporting

2 total gene-disease associations curated

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 0.99). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
58 unique Pathogenic / Likely Pathogenic· 259 VUS of 500 total submissions
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available
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GeneReview available — RBM20
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.29LOEUF
pLI 0.990
Z-score 5.22
OE 0.16 (0.090.29)
Highly constrained

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

Missense Constraint
1.67Z-score
OE missense 0.82 (0.760.88)
543 obs / 663.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.16 (0.090.29)
00.351.4
Missense OE0.82 (0.760.88)
00.61.4
Synonymous OE0.84
01.21.6
LoF obs/exp: 7 / 44.6Missense obs/exp: 543 / 663.8Syn Z: 2.10
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedRBM20-related hypertrophic cardiomyopathyOTHERAD
definitiveRBM20-related dilated cardiomyopathyOTHERAD
DN
0.3097th %ile
GOF
0.2796th %ile
LOF
0.76top 10%

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 · 1 literature citation · 91% of P/LP variants are LoF · LOEUF 0.29
GOF1 literature citation

Literature Evidence

GOFAnalysis of RBM20 RNA binding by eCLIP reveals a gain-of-function preference of mutant RBM20 for 3' UTR sequences that are shared with amyotrophic lateral sclerosis (ALS) and processing-body associated RNA binding proteins (FUS, DDX6).PMID:34732726
LOFA mutation outside the mutational hotspot of RBM20 results in haploinsufficiency of RBM20.PMID:27496873

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

500 submitted variants in ClinVar

Classification Summary

Pathogenic37
Likely Pathogenic21
VUS259
Likely Benign169
Benign3
Conflicting10
37
Pathogenic
21
Likely Pathogenic
259
VUS
169
Likely Benign
3
Benign
10
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
36
0
1
0
37
Likely Pathogenic
17
1
3
0
21
VUS
12
231
13
3
259
Likely Benign
0
14
43
112
169
Benign
0
2
1
0
3
Conflicting
10
Total6524861115499

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

View in ClinVar →

Protein Context — Lollipop Plot

RBM20 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold
Clinical Literature
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