TAFAZZIN

Chr XXLR

tafazzin, phospholipid-lysophospholipid transacylase

Also known as: BTHS, CMD3A, EFE, EFE2, G4.5, LVNCX, TAZ, Taz1

This acyltransferase remodels cardiolipin in the mitochondrial inner membrane, which is essential for proper mitochondrial function and respiratory chain complex assembly. Mutations cause Barth syndrome, an X-linked recessive disorder characterized by cardiomyopathy, skeletal myopathy, neutropenia, and growth delay that typically presents in infancy or early childhood. The gene shows moderate constraint against loss-of-function variants (LOEUF 0.485), reflecting its critical role in mitochondrial metabolism.

OMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismXLRLOEUF 0.481 OMIM phenotype
Clinical SummaryTAFAZZIN
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Gene-Disease Validity (ClinGen)
Barth syndrome · XLDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.73) — some intolerance to loss-of-function variants.
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.48LOEUF
pLI 0.726
Z-score 2.82
OE 0.15 (0.060.48)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
2.21Z-score
OE missense 0.44 (0.350.55)
54 obs / 122.9 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.15 (0.060.48)
00.351.4
Missense OE0.44 (0.350.55)
00.61.4
Synonymous OE0.83
01.21.6
LoF obs/exp: 2 / 13.0Missense obs/exp: 54 / 122.9Syn Z: 0.93

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

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