CST4 encodes a type 2 cystatin that functions as a cysteine protease inhibitor with strong activity against papain and ficin, and is highly expressed in saliva, tears, and other body fluids where it provides antibacterial and antiviral protection. Mutations in CST4 cause autosomal recessive amelogenesis imperfecta, a disorder affecting tooth enamel formation. The gene shows minimal constraint against loss-of-function variants, consistent with its recessive inheritance pattern.

Summary from RefSeq, UniProt
Research Assistant →
0
Active trials
13
Pubs (1 yr)
20
P/LP submissions
0%
P/LP missense
1.93
LOEUF
DN
Mechanism· predicted
Clinical SummaryCST4
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
20 unique Pathogenic / Likely Pathogenic· 29 VUS of 62 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.93LOEUF
pLI 0.000
Z-score -1.01
OE 1.46 (0.811.93)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-2.07Z-score
OE missense 1.63 (1.421.87)
138 obs / 84.5 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE1.46 (0.811.93)
00.351.4
Missense OE1.63 (1.421.87)
00.61.4
Synonymous OE1.58
01.21.6
LoF obs/exp: 8 / 5.5Missense obs/exp: 138 / 84.5Syn Z: -2.63
DN
0.7034th %ile
GOF
0.4480th %ile
LOF
0.2483th %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

62 submitted variants in ClinVar

Classification Summary

Pathogenic17
Likely Pathogenic3
VUS29
Likely Benign6
Benign6
17
Pathogenic
3
Likely Pathogenic
29
VUS
6
Likely Benign
6
Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
17
0
17
Likely Pathogenic
0
0
3
0
3
VUS
0
27
2
0
29
Likely Benign
0
3
0
3
6
Benign
0
3
1
2
6
Total03323561

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

View in ClinVar →

Protein Context — Lollipop Plot

CST4 · 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
Open Research Assistant →