The WDR64 protein is a component of the ciliary transition zone that regulates ciliary assembly and function. Mutations cause Jeune asphyxiating thoracic dystrophy and short-rib polydactyly syndrome, both autosomal recessive ciliopathies characterized by skeletal dysplasia, respiratory complications, and variable involvement of kidneys and other organs. These conditions typically present in the neonatal period with life-threatening thoracic restriction and may include polydactyly and renal cystic disease.

LOEUF 1.09
Clinical SummaryWDR64
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
67 unique Pathogenic / Likely Pathogenic· 168 VUS of 250 total submissions

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.09LOEUF
pLI 0.000
Z-score 0.99
OE 0.86 (0.691.09)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
0.86Z-score
OE missense 0.90 (0.830.97)
500 obs / 557.4 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.86 (0.691.09)
00.351.4
Missense OE0.90 (0.830.97)
00.61.4
Synonymous OE0.95
01.21.6
LoF obs/exp: 53 / 61.3Missense obs/exp: 500 / 557.4Syn Z: 0.53

ClinVar Variant Classifications

250 submitted variants in ClinVar

Classification Summary

Pathogenic64
Likely Pathogenic3
VUS168
Likely Benign6
64
Pathogenic
3
Likely Pathogenic
168
VUS
6
Likely Benign

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
0
64
0
64
Likely Pathogenic
0
0
3
0
3
VUS
0
109
59
0
168
Likely Benign
0
3
3
0
6
Benign
0
0
0
0
0
Total01121290241

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

View in ClinVar →

Protein Context — Lollipop Plot

WDR64 · 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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