TULP3

Chr 12AR

TUB like protein 3

Also known as: HRCDF, TUBL3

TULP3 encodes a negative regulator of Sonic hedgehog (Shh) signaling that controls trafficking of G protein-coupled receptors to primary cilia and binds phosphoinositide lipids. Mutations cause hepatorenocardiac degenerative fibrosis, a multisystem disorder affecting liver, kidney, and heart through autosomal recessive inheritance. The gene is extremely intolerant to loss-of-function variants (pLI near 1.0), indicating high evolutionary constraint.

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

Hepatorenocardiac degenerative fibrosisMIM #619902
AR
0
Active trials
13
Pubs (1 yr)
75
P/LP submissions
3%
P/LP missense
0.88
LOEUF
DN
Mechanism· predicted
Clinical SummaryTULP3
🧬
Gene-Disease Validity (ClinGen)
ciliopathy · ARStrong

Strong evidence — appropriate for clinical testing

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
72 unique Pathogenic / Likely Pathogenic· 81 VUS of 195 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.88LOEUF
pLI 0.000
Z-score 2.01
OE 0.55 (0.360.88)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.26Z-score
OE missense 0.96 (0.871.06)
283 obs / 295.4 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.55 (0.360.88)
00.351.4
Missense OE0.96 (0.871.06)
00.61.4
Synonymous OE1.06
01.21.6
LoF obs/exp: 13 / 23.5Missense obs/exp: 283 / 295.4Syn Z: -0.48
DN
0.75top 25%
GOF
0.5758th %ile
LOF
0.2775th %ile

The highest-scoring mechanism for this gene is dominant-negative.

DNprediction above median

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.

ClinVar Variant Classifications

195 submitted variants in ClinVar

Classification Summary

Pathogenic67
Likely Pathogenic5
VUS81
Likely Benign14
Benign3
Conflicting1
67
Pathogenic
5
Likely Pathogenic
81
VUS
14
Likely Benign
3
Benign
1
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
2
62
0
67
Likely Pathogenic
2
0
3
0
5
VUS
0
75
6
0
81
Likely Benign
0
10
1
3
14
Benign
0
2
1
0
3
Conflicting
1
Total589733171

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

View in ClinVar →

Protein Context — Lollipop Plot

TULP3 · 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.

Search ClinicalTrials.gov →
Clinical Literature
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