PCDH19

Chr XX-linked

protocadherin 19

Also known as: DEE9, EFMR, EIEE9

The protein functions as a calcium-dependent cell-adhesion protein primarily expressed in the brain. Mutations cause developmental and epileptic encephalopathy 9 through X-linked inheritance, with the unusual pattern of affecting heterozygous females more severely than hemizygous males due to X-inactivation creating cellular mosaicism. The disease mechanism predominantly involves loss-of-function, consistent with the gene's extreme intolerance to such variants.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismX-linkedLOEUF 0.131 OMIM phenotype
Clinical SummaryPCDH19
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Gene-Disease Validity (ClinGen)
X-linked complex neurodevelopmental disorder · XLDefinitive

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.

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.13LOEUF
pLI 1.000
Z-score 4.51
OE 0.00 (0.000.13)
Highly constrained

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

Missense Constraint
2.59Z-score
OE missense 0.67 (0.610.74)
336 obs / 499.0 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.00 (0.000.13)
00.351.4
Missense OE0.67 (0.610.74)
00.61.4
Synonymous OE1.02
01.21.6
LoF obs/exp: 0 / 23.7Missense obs/exp: 336 / 499.0Syn Z: -0.23
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitivePCDH19-related epileptic encephalopathy early infantileLOFmonoallelic_X_heterozygous
DN
0.5279th %ile
GOF
0.6542th %ile
LOF
0.67top 25%

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

LOFprediction above median · LOEUF 0.13
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. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

PCDH19 · 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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Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC