POLR3B

Chr 12ADAR

RNA polymerase III subunit B

Also known as: C128, CMT1I, HLD8, INMAP, RPC2

This gene encodes the second largest subunit of RNA polymerase III, which synthesizes transfer RNAs and small ribosomal RNAs and forms part of the enzyme's catalytic center. Mutations cause hypomyelinating leukodystrophy type 8 (with or without oligodontia and hypogonadism) and demyelinating Charcot-Marie-Tooth disease type 1I through both autosomal recessive and autosomal dominant inheritance patterns. The gene shows tolerance to loss-of-function variants, suggesting mutations can cause disease through multiple mechanisms depending on the specific variant type.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismAD/ARLOEUF 0.872 OMIM phenotypes
Clinical SummaryPOLR3B
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Gene-Disease Validity (ClinGen)
POLR3B-related disorder · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

2 total gene-disease associations curated

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
45 unique Pathogenic / Likely Pathogenic· 262 VUS of 497 total submissions
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GeneReview available — POLR3B
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Missense constrained — critical functional residues
LoF Constraint
0.87LOEUF
pLI 0.000
Z-score 2.46
OE 0.68 (0.540.87)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
3.20Z-score
OE missense 0.64 (0.590.70)
402 obs / 627.4 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios
LoF OE0.68 (0.540.87)
00.351.4
Missense OE0.64 (0.590.70)
00.61.4
Synonymous OE0.93
01.21.6
LoF obs/exp: 47 / 69.0Missense obs/exp: 402 / 627.4Syn Z: 0.80
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongPOLR3B-related neurodevelopmental disorder with or without seizures, ataxia, spasticity, and demyelinating neuropathyOTHERAD
definitivePOLR3B-related leukodystrophy, hypomyelinating with or without oligodontia and/or hypogonadotropic hypogonadismLOFAR
DN
0.7036th %ile
GOF
0.4973th %ile
LOF
0.4234th %ile

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

DNprediction above median
LOF58% of P/LP variants are LoF

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.

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

497 submitted variants in ClinVar

Classification Summary

Pathogenic16
Likely Pathogenic29
VUS262
Likely Benign108
Benign10
Conflicting22
16
Pathogenic
29
Likely Pathogenic
262
VUS
108
Likely Benign
10
Benign
22
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
8
4
4
0
16
Likely Pathogenic
18
9
2
0
29
VUS
2
225
26
9
262
Likely Benign
0
0
48
60
108
Benign
0
0
10
0
10
Conflicting
22
Total282389069447

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

View in ClinVar →

Protein Context — Lollipop Plot

POLR3B · 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
Open Research Assistant →
Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC