USP43

Chr 17

ubiquitin specific peptidase 43

The USP43 protein is a deubiquitinating enzyme that cleaves ubiquitin from target proteins and processes polyubiquitin precursors, and may also have ISG15-specific peptidase activity involved in DNA repair processes. This gene is highly constrained against loss-of-function variants (LOEUF 0.398), but no definitive human disease associations have been established for USP43 mutations. Further research is needed to determine the clinical significance of variants in this gene.

ResearchSummary from RefSeq, UniProt
MultiplemechanismLOEUF 0.40
Clinical SummaryUSP43
Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.24) despite low pLI — interpret in context.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.40LOEUF
pLI 0.164
Z-score 4.76
OE 0.24 (0.150.40)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
1.58Z-score
OE missense 0.81 (0.750.88)
468 obs / 574.8 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.24 (0.150.40)
00.351.4
Missense OE0.81 (0.750.88)
00.61.4
Synonymous OE0.87
01.21.6
LoF obs/exp: 11 / 45.7Missense obs/exp: 468 / 574.8Syn Z: 1.65
DN
0.7230th %ile
GOF
0.6735th %ile
LOF
0.3940th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative and gain-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

DNprediction above median
GOFprediction 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

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

USP43 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

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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