SH3BP4

Chr 2

SH3 domain binding protein 4

Also known as: BOG25, TTP

This protein regulates clathrin-mediated endocytosis of transferrin receptors and acts as a negative regulator of mTOR signaling by preventing Rag GTPase complex activation, thereby controlling cell growth, proliferation, and autophagy. Mutations cause autosomal recessive neurodevelopmental disorder with progressive microcephaly, seizures, and brain atrophy. The gene shows tolerance to loss-of-function variants (low pLI score), consistent with the recessive inheritance pattern observed clinically.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
4
Pubs (1 yr)
48
P/LP submissions
0%
P/LP missense
0.57
LOEUF
Mechanism
Clinical SummarySH3BP4
Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.34) despite low pLI — interpret in context.
📋
ClinVar Variants
48 unique Pathogenic / Likely Pathogenic· 140 VUS of 225 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.57LOEUF
pLI 0.003
Z-score 3.35
OE 0.34 (0.210.57)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
0.63Z-score
OE missense 0.93 (0.860.99)
539 obs / 581.6 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.34 (0.210.57)
00.351.4
Missense OE0.93 (0.860.99)
00.61.4
Synonymous OE1.05
01.21.6
LoF obs/exp: 10 / 29.7Missense obs/exp: 539 / 581.6Syn Z: -0.64

ClinVar Variant Classifications

225 submitted variants in ClinVar

Classification Summary

Pathogenic47
Likely Pathogenic1
VUS140
Likely Benign16
Benign7
47
Pathogenic
1
Likely Pathogenic
140
VUS
16
Likely Benign
7
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
47
0
47
Likely Pathogenic
0
0
1
0
1
VUS
0
132
8
0
140
Likely Benign
0
8
1
7
16
Benign
0
1
0
6
7
Total01415713211

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

View in ClinVar →

Protein Context — Lollipop Plot

SH3BP4 · 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.

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Clinical Literature
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