TBCK

Chr 4AR

TBC1 domain containing kinase

Also known as: FERRY1, Fy-1, HSPC302, IHPRF3, TBCKL

The protein contains a kinase domain and regulates mTOR signaling pathway to control actin organization, cell growth, and cell proliferation. Biallelic mutations cause hypotonia, infantile, with psychomotor retardation and characteristic facies 3, inherited in an autosomal recessive pattern. The pathogenic mechanism involves loss of function, disrupting normal mTOR-mediated cellular processes essential for neuronal development.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismARLOEUF 0.871 OMIM phenotype
Clinical SummaryTBCK
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Gene-Disease Validity (ClinGen)
syndromic complex neurodevelopmental disorder · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
137 unique Pathogenic / Likely Pathogenic· 369 VUS of 982 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 — TBCK
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.87LOEUF
pLI 0.000
Z-score 2.28
OE 0.65 (0.480.87)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
0.50Z-score
OE missense 0.93 (0.861.01)
418 obs / 448.0 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.65 (0.480.87)
00.351.4
Missense OE0.93 (0.861.01)
00.61.4
Synonymous OE0.99
01.21.6
LoF obs/exp: 31 / 48.0Missense obs/exp: 418 / 448.0Syn Z: 0.07
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveTBCK-related severe infantile syndromic encephalopathyLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.6936th %ile
GOF
0.6540th %ile
LOF
0.2872th %ile

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

982 submitted variants in ClinVar

Classification Summary

Pathogenic76
Likely Pathogenic61
VUS369
Likely Benign366
Benign72
Conflicting16
76
Pathogenic
61
Likely Pathogenic
369
VUS
366
Likely Benign
72
Benign
16
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
45
0
31
0
76
Likely Pathogenic
52
1
8
0
61
VUS
2
324
40
3
369
Likely Benign
0
10
190
166
366
Benign
0
5
60
7
72
Conflicting
16
Total99340329176960

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

View in ClinVar →

Protein Context — Lollipop Plot

TBCK · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
Open Research Assistant →