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10 sources retrieved · Most recent: April 2026 · Index updated 14 days ago
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PIGH
phosphatidylinositol glycan anchor biosynthesis class H
Chromosome 14 · 14q24.1
NCBI Gene: 5283Ensembl: ENSG00000100564.10HGNC: HGNC:8964UniProt: H0YLY9
30PubMed Papers
21Diseases
0Drugs
2Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
protein bindingGPI anchor biosynthetic processendoplasmic reticulum membraneglycosylphosphatidylinositol-N-acetylglucosaminyltransferase (GPI-GnT) complexglycosylphosphatidylinositol biosynthesis defect 17epilepsyFebrile seizure (within the age range of 3 months to 6 years)disorder of GPI anchor biosynthesis
✦AI Summary

PIGH (phosphatidylinositol glycan anchor biosynthesis class H) encodes an essential component of the glycosylphosphatidylinositol N-acetylglucosaminyltransferase (GPI-GnT) complex, catalyzing the transfer of N-acetylglucosamine to phosphatidylinositol in the first step of GPI anchor biosynthesis 1. GPI anchors are glycolipid modifications added to over one hundred human proteins critical for embryogenesis and neurological function 1. PIGH mutations cause glycosylphosphatidylinositol biosynthesis defect 17, a rare autosomal recessive disorder with heterogeneous clinical severity. Mild cases present with developmental delay, hypotonia, and autism without epilepsy 1, while severe cases feature intractable seizures, profound psychomotor retardation, microcephaly, skeletal anomalies including bone fractures and scoliosis, and delayed myelination 2. The p.(Arg163Trp) variant associates with particularly severe manifestations 2. Pathogenic variants range from missense mutations to start-codon disruptions causing truncation at downstream codons 3. Surface expression analysis reveals decreased GPI-anchored proteins on patient cells 12. Additionally, PIGH expression is epigenetically silenced in some B-lymphoblastic leukemia cases, contributing to CD52 loss and alemtuzumab resistance 4. These findings establish PIGH as critical for proper GPI biosynthesis and neurological development.

Sources cited
1
PIGH encodes a component of GPI-GnT complex; mutations cause developmental delay, autism, hypotonia, and GPI deficiency
PMID: 29603516
2
PIGH deficiency presents with variable severity; p.(Arg163Trp) variant associates with severe neurodevelopmental and skeletal manifestations including seizures, fractures, and scoliosis
PMID: 33156547
3
Homozygous c.1A > T start-codon variant in PIGH causes epilepsy, microcephaly, developmental delay, and defective GPI-anchor biogenesis
PMID: 29573052
4
PIGH expression is epigenetically silenced in B-lymphoblastic leukemia, leading to GPI-anchor deficiency and CD52 loss
PMID: 30370942
⚠Limited data available — This gene has 4 indexed publications. Summary and analysis may be incomplete.
Disease Associationsⓘ21
glycosylphosphatidylinositol biosynthesis defect 17Open Targets
0.73Strong
epilepsyOpen Targets
0.37Weak
disorder of GPI anchor biosynthesisOpen Targets
0.37Weak
Febrile seizure (within the age range of 3 months to 6 years)Open Targets
0.37Weak
neurodegenerative diseaseOpen Targets
0.30Weak
genetic disorderOpen Targets
0.19Weak
Familial prostate cancerOpen Targets
0.11Weak
prostate cancerOpen Targets
0.11Weak
acute lymphoblastic leukemiaOpen Targets
0.06Suggestive
hypertriglyceridemia 2Open Targets
0.04Suggestive
pancreatic triacylglycerol lipase deficiencyOpen Targets
0.03Suggestive
isolated hyperchlorhidrosisOpen Targets
0.03Suggestive
ACPA-positive rheumatoid arthritisOpen Targets
0.03Suggestive
nonpapillary renal cell carcinomaOpen Targets
0.03Suggestive
liver diseaseOpen Targets
0.02Suggestive
hyperinsulinemic hypoglycemia, familial, 4Open Targets
0.01Suggestive
Global developmental delayOpen Targets
0.01Suggestive
type 1 diabetes mellitusOpen Targets
0.01Suggestive
arthrogryposisOpen Targets
0.01Suggestive
microcephalyOpen Targets
0.01Suggestive
Glycosylphosphatidylinositol biosynthesis defect 17UniProt
Pathogenic Variants2
NM_004569.5(PIGH):c.307T>C (p.Ser103Pro)Pathogenic
Glycosylphosphatidylinositol biosynthesis defect 17
★★☆☆2020→ Residue 103
NM_004569.5(PIGH):c.1A>T (p.Met1Leu)Pathogenic
Glycosylphosphatidylinositol biosynthesis defect 17
☆☆☆☆2018→ Residue 1
View on ClinVar ↗
Related Genes
PGAP4Shared pathway100%PIGZShared pathway100%CWH43Shared pathway100%PIGXShared pathway100%PIGPProtein interaction100%PIGYProtein interaction100%
Tissue Expression6 tissues
Bone Marrow
100%
Ovary
55%
Liver
52%
Brain
50%
Lung
36%
Heart
23%
Gene Interaction Network
Click a node to explore
PIGHPGAP4PIGZCWH43PIGXPIGPPIGY
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted · UniProt Q14442
View on AlphaFold ↗
Constraintⓘ
LOEUFⓘ
1.78LoF Tolerant
pLIⓘ
0.00Tolerant
Observed/Expected LoF1.09 [0.65–1.78]
RankingsWhere PIGH stands among ~20K protein-coding genes
  • #12,003of 20,598
    Most Researched30
  • #4,519of 5,498
    Most Pathogenic Variants2
  • #16,468of 17,882
    Most Constrained (LOEUF)1.78
Genes detectedPIGH
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
A PIGH mutation leading to GPI deficiency is associated with developmental delay and autism.
PMID: 29603516
Hum Mutat · 2018
1.00
2
PIGH deficiency can be associated with severe neurodevelopmental and skeletal manifestations.
PMID: 33156547
Clin Genet · 2021
0.90
3
Pathogenic glycosyltransferase genes and potential therapeutic drugs in pressure overload-induced heart failure.
PMID: 40903443
ESC Heart Fail · 2025
0.80
4
Expanding the phenotype of PIGP deficiency to multiple congenital anomalies-hypotonia-seizures syndrome.
PMID: 37125481
Clin Genet · 2023
0.70
5
A homozygous variant disrupting the PIGH start-codon is associated with developmental delay, epilepsy, and microcephaly.
PMID: 29573052
Hum Mutat · 2018
0.60