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10 sources retrieved · Most recent: April 2026 · Index updated 15 days ago
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B4GAT1
beta-1,4-glucuronyltransferase 1
Chromosome 11 · 11q13.2
NCBI Gene: 11041Ensembl: ENSG00000174684.8HGNC: HGNC:15685UniProt: O43505
49PubMed Papers
21Diseases
0Drugs
6Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
Golgi apparatuspoly-N-acetyllactosamine biosynthetic processprotein bindingglucuronosyltransferase activitymuscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A13muscular dystrophy-dystroglycanopathy, type Acongenital muscular dystrophymuscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A1
✦AI Summary

B4GAT1 (beta-1,4-glucuronyltransferase 1) is a Golgi-localized glycosyltransferase essential for O-mannosyl glycosylation of alpha-dystroglycan (α-DG) 1. The enzyme catalyzes transfer of glucuronic acid onto a xylose acceptor, generating a glucuronyl-β1,4-xylose disaccharide primer 1 that is subsequently elongated by LARGE1 to form phosphorylated O-mannosyl glycans 2. These glycan modifications enable α-DG to bind laminin G-like domain-containing extracellular matrix proteins with high affinity, maintaining tissue integrity and basement membrane organization 1. B4GAT1 localizes to the trans-Golgi network alongside other DG-modifying enzymes 2, functioning at distinct subcellular compartments from its primary localization sites 2. Mutations in B4GAT1 cause muscular dystrophy-dystroglycanopathy type A13, presenting with congenital muscular dystrophy, severe ventriculomegaly, and cerebellar hypoplasia 3. Notably, B4GAT1 downregulation in various human tumors correlates with impaired α-DG glycosylation and disrupted cell-matrix interactions 4. Additionally, B4GAT1 downregulation appears in idiopathic normal pressure hydrocephalus 5, suggesting possible shared molecular mechanisms with congenital hydrocephalus. The gene's role in axon guidance through α-DG O-mannosylation remains important for proper neuromuscular development.

Sources cited
1
B4GAT1 is a xylose β1,4-glucuronyltransferase that catalyzes synthesis of glucuronic acid β1,4-xylose disaccharide primer for α-DG O-mannosyl glycosylation
PMID: 25279699
2
B4GAT1 localizes to the trans-Golgi network and functions in DG-specific O-glycan modification in the Golgi apparatus
PMID: 40324206
3
B4GAT1 mutations cause congenital muscular dystrophy-dystroglycanopathy type A13 with ventriculomegaly and cerebellar hypoplasia
PMID: 34587870
4
B4GAT1 is downregulated in multiple human tumors, correlating with impaired α-DG glycosylation and cancer progression
PMID: 36494657
5
B4GAT1 downregulation is associated with idiopathic normal pressure hydrocephalus, suggesting shared pathophysiology with congenital hydrocephalus
PMID: 39118132
Disease Associationsⓘ21
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A13Open Targets
0.77Strong
muscular dystrophy-dystroglycanopathy, type AOpen Targets
0.60Moderate
congenital muscular dystrophyOpen Targets
0.37Weak
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A1Open Targets
0.26Weak
genetic disorderOpen Targets
0.19Weak
handednessOpen Targets
0.17Weak
goutOpen Targets
0.16Weak
46,XX gonadal dysgenesisOpen Targets
0.12Weak
genetic non-acquired premature ovarian failureOpen Targets
0.12Weak
attention deficit hyperactivity disorderOpen Targets
0.09Suggestive
diverticular diseaseOpen Targets
0.08Suggestive
attention deficit-hyperactivity disorder 8Open Targets
0.07Suggestive
hereditary attention deficit-hyperactivity disorderOpen Targets
0.07Suggestive
intellectual disability, autosomal recessive 59Open Targets
0.07Suggestive
movement disorderOpen Targets
0.07Suggestive
schizophrenia 15Open Targets
0.07Suggestive
Phelan-McDermid syndromeOpen Targets
0.05Suggestive
autismOpen Targets
0.05Suggestive
Tourette syndromeOpen Targets
0.05Suggestive
X-linked non-syndromic intellectual disabilityOpen Targets
0.04Suggestive
Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A13UniProt
Pathogenic Variants6
NM_006876.3(B4GAT1):c.1217C>T (p.Ala406Val)Likely pathogenic
not provided
★☆☆☆2021→ Residue 406
NM_006876.3(B4GAT1):c.1168A>G (p.Asn390Asp)Likely pathogenic
not provided
★☆☆☆2021→ Residue 390
NM_006876.3(B4GAT1):c.864T>A (p.Tyr288Ter)Pathogenic
Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A13
★☆☆☆2021→ Residue 288
NM_006876.3(B4GAT1):c.1179_1181del (p.Asn393del)Likely pathogenic
not provided
★☆☆☆2017→ Residue 393
NM_006876.3(B4GAT1):c.1207G>T (p.Glu403Ter)Pathogenic
Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A13
☆☆☆☆2021→ Residue 403
NM_006876.3(B4GAT1):c.821_822insTT (p.Glu274fs)Pathogenic
Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A1|Muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A13
☆☆☆☆2018→ Residue 274
View on ClinVar ↗
Related Genes
FKRPProtein interaction100%POMGNT1Protein interaction99%POMT2Protein interaction99%CRPPAProtein interaction98%DAG1Protein interaction98%POMKProtein interaction82%
Tissue Expression6 tissues
Brain
100%
Heart
46%
Ovary
30%
Liver
17%
Lung
11%
Bone Marrow
3%
Gene Interaction Network
Click a node to explore
B4GAT1FKRPPOMGNT1POMT2CRPPADAG1POMK
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted · UniProt O43505
View on AlphaFold ↗
Constraintⓘ
LOEUFⓘ
0.90LoF Tolerant
pLIⓘ
0.00Tolerant
Observed/Expected LoF0.61 [0.42–0.90]
RankingsWhere B4GAT1 stands among ~20K protein-coding genes
  • #8,914of 20,598
    Most Researched49
  • #3,398of 5,498
    Most Pathogenic Variants6
  • #8,091of 17,882
    Most Constrained (LOEUF)0.90
Genes detectedB4GAT1
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort.
PMID: 34602496
J Neuromuscul Dis · 2022
1.00
2
PMID: 34587870
Fetal Pediatr Pathol · 2022
0.90
3
Involvement of muscle satellite cell dysfunction in neuromuscular disorders: Expanding the portfolio of satellite cell-opathies.
PMID: 35302338
Eur J Transl Myol · 2022
0.80
4
B4GALT1 promotes immune escape by regulating the expression of PD-L1 at multiple levels in lung adenocarcinoma.
PMID: 37303063
J Exp Clin Cancer Res · 2023
0.70
5
Molecular signatures of normal pressure hydrocephalus: a large-scale proteomic analysis of cerebrospinal fluid.
PMID: 39118132
Fluids Barriers CNS · 2024
0.60