WDFY3

Chr 4AD

WD repeat and FYVE domain containing 3

Also known as: ALFY, BCHS, MCPH18, ZFYVE25

The protein encoded by this gene binds phosphatidylinositol 3-phosphate and functions as an adapter protein that links aggregated proteins to the autophagy machinery for selective clearance, playing an essential role in brain development including formation of axonal tracts and major forebrain commissures. Mutations cause primary microcephaly 18 with autosomal dominant inheritance. This gene is highly constrained against loss-of-function variants (pLI = 1, LOEUF = 0.071), consistent with its essential role in early brain development.

OMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismADLOEUF 0.071 OMIM phenotype
Clinical SummaryWDFY3
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Gene-Disease Validity (ClinGen)
syndromic intellectual disability · ADDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
20 unique Pathogenic / Likely Pathogenic· 88 VUS of 119 total submissions
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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

Dual constrained — LoF & missense intolerant
LoF Constraint
0.07LOEUF
pLI 1.000
Z-score 12.10
OE 0.04 (0.020.07)
Highly constrained

Among the most LoF-intolerant genes (~top 3%)

Missense Constraint
5.82Z-score
OE missense 0.62 (0.590.65)
1176 obs / 1887.2 exp
Constrained

Extremely missense-constrained (top ~0.01%)

Observed / Expected Ratios
LoF OE0.04 (0.020.07)
00.351.4
Missense OE0.62 (0.590.65)
00.61.4
Synonymous OE1.02
01.21.6
LoF obs/exp: 7 / 184.3Missense obs/exp: 1176 / 1887.2Syn Z: -0.36
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
strongWDFY3-related primary microcephaly or macrocephaly with developmental delayLOFAD
DN
0.2599th %ile
GOF
0.2895th %ile
LOF
0.75top 10%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median · 1 literature citation · 25% of P/LP variants are LoF · LOEUF 0.07

Literature Evidence

LOFConsequently, we propose that in humans WDFY3 loss-of-function variants lead to macrocephaly via downregulation of the Wnt pathway.PMID:31327001

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

119 submitted variants in ClinVar

Classification Summary

Pathogenic17
Likely Pathogenic3
VUS88
Likely Benign8
Conflicting3
17
Pathogenic
3
Likely Pathogenic
88
VUS
8
Likely Benign
3
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
0
14
0
17
Likely Pathogenic
2
1
0
0
3
VUS
3
81
3
1
88
Likely Benign
0
8
0
0
8
Benign
0
0
0
0
0
Conflicting
3
Total890171119

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

View in ClinVar →

Protein Context — Lollipop Plot

WDFY3 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
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