ZBTB48

Chr 1

zinc finger and BTB domain containing 48

Also known as: HKR3, TZAP, ZNF855, pp9964

The protein directly binds telomeric DNA and regulates telomere length by initiating telomere trimming to prevent accumulation of aberrantly long telomeres, while also functioning as a transcription regulator for genes including the tumor suppressor ARF. Mutations cause autosomal dominant intellectual disability with short stature, behavioral problems, and distinctive facial features. This gene is highly intolerant to loss-of-function mutations, suggesting complete loss of protein function is likely incompatible with normal development.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
3
Pubs (1 yr)
52
P/LP submissions
0%
P/LP missense
0.64
LOEUF
DN
Mechanism· predicted
Clinical SummaryZBTB48
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
52 unique Pathogenic / Likely Pathogenic· 81 VUS of 148 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.64LOEUF
pLI 0.000
Z-score 3.07
OE 0.40 (0.250.64)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
1.21Z-score
OE missense 0.83 (0.760.91)
348 obs / 417.3 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.40 (0.250.64)
00.351.4
Missense OE0.83 (0.760.91)
00.61.4
Synonymous OE1.16
01.21.6
LoF obs/exp: 12 / 30.2Missense obs/exp: 348 / 417.3Syn Z: -1.63
DN
0.6649th %ile
GOF
0.5465th %ile
LOF
0.3745th %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

148 submitted variants in ClinVar

Classification Summary

Pathogenic51
Likely Pathogenic1
VUS81
Likely Benign5
Benign1
51
Pathogenic
1
Likely Pathogenic
81
VUS
5
Likely Benign
1
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
51
0
51
Likely Pathogenic
0
0
1
0
1
VUS
0
75
6
0
81
Likely Benign
0
2
2
1
5
Benign
0
0
1
0
1
Total077611139

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

View in ClinVar →

Protein Context — Lollipop Plot

ZBTB48 · 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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Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC