PIK3C3

Chr 18

phosphatidylinositol 3-kinase catalytic subunit type 3

Also known as: VPS34, Vps34, hVps34

PIK3C3 encodes a catalytic subunit that generates phosphatidylinositol 3-phosphate in different PI3K complexes, mediating autophagosome formation, endosomal trafficking, and cytokinesis. Mutations cause autosomal recessive neurodevelopmental disorders with seizures and intellectual disability, often with early childhood onset. The gene is highly constrained against loss-of-function variants in the population.

Summary from RefSeq, UniProt
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0
Active trials
38
Pubs (1 yr)
0
P/LP submissions
P/LP missense
0.67
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryPIK3C3
Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.67LOEUF
pLI 0.000
Z-score 3.55
OE 0.48 (0.350.67)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
2.44Z-score
OE missense 0.68 (0.630.75)
325 obs / 474.6 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.48 (0.350.67)
00.351.4
Missense OE0.68 (0.630.75)
00.61.4
Synonymous OE1.05
01.21.6
LoF obs/exp: 26 / 54.2Missense obs/exp: 325 / 474.6Syn Z: -0.52
DN
0.6842th %ile
GOF
0.6834th %ile
LOF
0.3357th %ile

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

GOFprediction above median
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

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

PIK3C3 · 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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