TGFBR2

Chr 3AD

transforming growth factor beta receptor 2

Also known as: AAT3, FAA3, LDS1B, LDS2, LDS2B, MFS2, RIIC, TAAD2

The transmembrane serine/threonine kinase receptor forms complexes with TGF-beta cytokines and TGFBR1 to regulate cell proliferation, differentiation, wound healing, and immune responses through SMAD-dependent signaling pathways. Mutations cause Loeys-Dietz syndrome 2, a connective tissue disorder affecting the cardiovascular system, and hereditary nonpolyposis colorectal cancer type 6, both following autosomal dominant inheritance. The gene shows moderate constraint against loss-of-function variants (LOEUF 0.52), reflecting its essential role in multiple developmental and physiological processes.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismADLOEUF 0.523 OMIM phenotypes
Clinical SummaryTGFBR2
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Gene-Disease Validity (ClinGen)
Loeys-Dietz syndrome 2 · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.26) despite low pLI — interpret in context.
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ClinVar Variants
36 unique Pathogenic / Likely Pathogenic· 293 VUS of 495 total submissions
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GeneReview available — TGFBR2
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.52LOEUF
pLI 0.125
Z-score 3.26
OE 0.26 (0.140.52)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
2.24Z-score
OE missense 0.65 (0.580.73)
215 obs / 329.6 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.26 (0.140.52)
00.351.4
Missense OE0.65 (0.580.73)
00.61.4
Synonymous OE1.07
01.21.6
LoF obs/exp: 6 / 22.8Missense obs/exp: 215 / 329.6Syn Z: -0.60
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveTGFBR2-related Loeys-Dietz syndromeLOFAD
DN
0.6358th %ile
GOF
0.7125th %ile
LOF
0.3744th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function, dominant-negative 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 · 83% of P/LP are missense
DNprediction above median
LOF1 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

GOFThe fact that the non-immune manifestations of the gain-of-function mutations of TGFBR1 and TGFBR2 are similar to the those of dominant negative mutations of STAT3 provide a clue to elucidate molecular mechanisms of non-immune manifestations of hyper-IgE syndrome.PMID:34419355
LOFHeterozygous loss-of-function mutations in TGF-? receptors 1 and 2 (TGFBR1 and TGFBR2) cause LDS, but TGF-? signaling is activated in the aorta (referred to as the TGF-? paradox) by mechanisms yet to be elucidated.PMID:30037098

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

495 submitted variants in ClinVar

Classification Summary

Pathogenic15
Likely Pathogenic21
VUS293
Likely Benign150
Benign3
Conflicting6
15
Pathogenic
21
Likely Pathogenic
293
VUS
150
Likely Benign
3
Benign
6
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
11
4
0
15
Likely Pathogenic
2
19
0
0
21
VUS
20
253
20
0
293
Likely Benign
1
2
43
104
150
Benign
0
1
2
0
3
Conflicting
6
Total2328669104488

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

View in ClinVar →

Protein Context — Lollipop Plot

TGFBR2 · 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 →
Key Publications
Landmark & review papers · by relevance
PubMed
Top 5 results · since 2015Search PubMed ↗