Raras
Buscar doenças, sintomas, genes...
Alteração da proteína O-glicosilação
ORPHA:309447CID-11 · 5C54.1DOENÇA RARA

Uma doença que tem sua origem em uma alteração na glicosilação O-ligada de proteínas, que é o processo de adicionar açúcares a elas de uma forma específica.

Mantido por Agente Raras·Colaborar como especialista →

Introdução

O que você precisa saber de cara

📋

Uma doença que tem sua origem em uma alteração na glicosilação O-ligada de proteínas, que é o processo de adicionar açúcares a elas de uma forma específica.

Publicações científicas
1 artigos
Último publicado: 2017 Apr
🏥
SUS: Sem cobertura SUSScore: 0%
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Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🦴
Ossos e articulações
52 sintomas
💪
Músculos
46 sintomas
🧠
Neurológico
40 sintomas
👁️
Olhos
29 sintomas
😀
Face
23 sintomas
❤️
Coração
18 sintomas

+ 157 sintomas em outras categorias

Características mais comuns

Meningocele
Agenesia do incisivo lateral maxilar
Fraqueza dos músculos do pescoço
Agenesia do corpo caloso
Catarata do desenvolvimento
Intolerância ao exercício
430sintomas
Sem dados (430)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 430 características clínicas mais associadas, ordenadas por frequência.

Meningocele
Agenesia do incisivo lateral maxilarAgenesis of maxillary lateral incisor
Fraqueza dos músculos do pescoçoNeck muscle weakness
Agenesia do corpo calosoAgenesis of corpus callosum
Catarata do desenvolvimentoDevelopmental cataract

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa5
Total histórico1PubMed
Últimos 10 anos200publicações
Pico202555 papers
Linha do tempo
2021Hoje · 2026📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

20 genes identificados com associação a esta condição.

POGLUT1Protein O-glucosyltransferase 1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Dual specificity glycosyltransferase that catalyzes the transfer of glucose and xylose from UDP-glucose and UDP-xylose, respectively, to a serine residue found in the consensus sequence of C-X-S-X-P-C (PubMed:21081508, PubMed:21490058, PubMed:21949356, PubMed:27807076, PubMed:28775322). Specifically targets extracellular EGF repeats of protein such as CRB2, F7, F9 and NOTCH2 (PubMed:21081508, PubMed:21490058, PubMed:21949356, PubMed:27807076, PubMed:28775322). Acts as a positive regulator of Not

LOCALIZAÇÃO

Endoplasmic reticulum lumen

VIAS BIOLÓGICAS (1)
Pre-NOTCH Processing in the Endoplasmic Reticulum
MECANISMO DE DOENÇA

Dowling-Degos disease 4

A form of Dowling-Degos disease, a genodermatosis manifesting with postpubertal reticulate hyperpigmentation that is progressive and disfiguring, and small hyperkeratotic dark brown papules that affect mainly the flexures and great skin folds. Patients usually show no abnormalities of the hair or nails. DDD4 is characterized by prominent involvement of non-flexural skin areas.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
18.8 TPM
Linfócitos
18.3 TPM
Cervix Ectocervix
17.1 TPM
Cervix Endocervix
16.6 TPM
Baço
16.2 TPM
OUTRAS DOENÇAS (3)
autosomal recessive limb-girdle muscular dystrophy type 2R1Dowling-Degos disease 4Dowling-Degos disease
HGNC:22954UniProt:Q8NBL1
TBX6T-box transcription factor TBX6Candidate gene tested inTolerante
FUNÇÃO

T-box transcription factor that plays an essential role in the determination of the fate of axial stem cells: neural vs mesodermal. Acts in part by down-regulating, a specific enhancer (N1) of SOX2, to inhibit neural development. Seems to play also an essential role in left/right axis determination and acts through effects on Notch signaling around the node as well as through an effect on the morphology and motility of the nodal cilia (By similarity)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (3)
Formation of paraxial mesodermSomitogenesisFormation of the posterior neural plate
MECANISMO DE DOENÇA

Spondylocostal dysostosis 5

A rare condition of variable severity characterized by vertebral and costal anomalies. The main feature include dwarfism, vertebral fusion, hemivertebrae, posterior rib fusion, reduced rib number, and other rib malformations. SCDO5 inheritance can be autosomal dominant or recessive.

EXPRESSÃO TECIDUAL(Ubíquo)
Esôfago - Mucosa
10.7 TPM
Vagina
7.6 TPM
Bladder
4.0 TPM
Tireoide
3.8 TPM
Skin Not Sun Exposed Suprapubic
3.4 TPM
OUTRAS DOENÇAS (3)
spondylocostal dysostosis 5autosomal recessive spondylocostal dysostosisautosomal dominant spondylocostal dysostosis
HGNC:11605UniProt:O95947
CRPPAD-ribitol-5-phosphate cytidylyltransferaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Cytidylyltransferase required for protein O-linked mannosylation (PubMed:22522420, PubMed:22522421, PubMed:26687144, PubMed:26923585, PubMed:27130732, PubMed:27601598). Catalyzes the formation of CDP-ribitol nucleotide sugar from D-ribitol 5-phosphate (PubMed:26687144, PubMed:26923585, PubMed:27130732). CDP-ribitol is a substrate of FKTN during the biosynthesis of the phosphorylated O-mannosyl trisaccharide (N-acetylgalactosamine-beta-3-N-acetylglucosamine-beta-4-(phosphate-6-)mannose), a carboh

LOCALIZAÇÃO

Cytoplasm, cytosol

VIAS BIOLÓGICAS (1)
Matriglycan biosynthesis on DAG1
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A7

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with cobblestone lissencephaly and other brain anomalies, eye malformations, profound intellectual disability, and death usually in the first years of life. Included diseases are the more severe Walker-Warburg syndrome and the slightly less severe muscle-eye-brain disease.

OUTRAS DOENÇAS (5)
autosomal recessive limb-girdle muscular dystrophy type 2Umuscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A, 7muscle-eye-brain diseasemuscular dystrophy-dystroglycanopathy, type A
HGNC:37276UniProt:A4D126
KRT5Keratin, type II cytoskeletal 5Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Required for the formation of keratin intermediate filaments in the basal epidermis and maintenance of the skin barrier in response to mechanical stress (By similarity). Regulates the recruitment of Langerhans cells to the epidermis, potentially by modulation of the abundance of macrophage chemotactic cytokines, macrophage inflammatory cytokines and CTNND1 localization in keratinocytes (By similarity)

LOCALIZAÇÃO

Cytoplasm

VIAS BIOLÓGICAS (7)
Type I hemidesmosome assemblyKeratinizationFormation of the cornified envelopeDevelopmental Lineage of Mammary Stem CellsDevelopmental Lineage of Mammary Gland Luminal Epithelial Cells
MECANISMO DE DOENÇA

Epidermolysis bullosa simplex 2A, generalized severe

A form of epidermolysis bullosa simplex, a group of skin fragility disorders characterized by skin blistering due to cleavage within the basal layer of keratinocytes, and erosions caused by minor mechanical trauma. There is a broad spectrum of clinical severity ranging from minor blistering on the feet, to subtypes with extracutaneous involvement and a lethal outcome. EBS2A is an autosomal dominant, severe form characterized by extensive intraepidermal blistering from the time of birth with herpetiform marginal spreading and central healing. Oral mucosal involvement, nail dystrophy, onychogryposis, formation of milia, and palmoplantar hyperkeratosis are common features.

EXPRESSÃO TECIDUAL(Ubíquo)
Esôfago - Mucosa
7391.8 TPM
Skin Not Sun Exposed Suprapubic
5833.1 TPM
Skin Sun Exposed Lower leg
5020.4 TPM
Vagina
3291.8 TPM
Glândula salivar
340.6 TPM
OUTRAS DOENÇAS (11)
epidermolysis bullosa simplex 2F, with mottled pigmentationepidermolysis bullosa simplex 2B, generalized intermediateepidermolysis bullosa simplex 2E, with migratory circinate erythemaDowling-Degos disease 1
HGNC:6442UniProt:P13647
PSENENGamma-secretase subunit PEN-2Candidate gene tested inRestrito
FUNÇÃO

Essential subunit of the gamma-secretase complex, an endoprotease complex that catalyzes the intramembrane cleavage of integral membrane proteins such as Notch receptors and APP (amyloid-beta precursor protein) (PubMed:12522139, PubMed:12679784, PubMed:12740439, PubMed:12763021, PubMed:24941111, PubMed:30598546, PubMed:30630874). The gamma-secretase complex plays a role in Notch and Wnt signaling cascades and regulation of downstream processes via its role in processing key regulatory proteins,

LOCALIZAÇÃO

Endoplasmic reticulum membraneGolgi apparatus, Golgi stack membraneCell membraneMembrane

VIAS BIOLÓGICAS (10)
Activated NOTCH1 Transmits Signal to the NucleusNOTCH3 Activation and Transmission of Signal to the NucleusNOTCH4 Activation and Transmission of Signal to the NucleusNoncanonical activation of NOTCH3NOTCH2 Activation and Transmission of Signal to the Nucleus
MECANISMO DE DOENÇA

Acne inversa, familial, 2, with or without Dowling-Degos disease

An autosomal dominant form of acne inversa, a chronic relapsing inflammatory disease of the hair follicles characterized by recurrent draining sinuses, painful skin abscesses, and disfiguring scars. Manifestations typically appear after puberty. Some ACNINV2 patients also exhibit reticulate hyperpigmentation consistent with Dowling-Degos disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
189.4 TPM
Tireoide
64.5 TPM
Cervix Endocervix
59.0 TPM
Pituitária
56.5 TPM
Sangue
55.5 TPM
OUTRAS DOENÇAS (2)
acne inversa, familial, 2Dowling-Degos disease
HGNC:30100UniProt:Q9NZ42
POFUT1GDP-fucose protein O-fucosyltransferase 1Disease-causing germline mutation(s) inRestrito
FUNÇÃO

Catalyzes the reaction that attaches fucose through an O-glycosidic linkage to a conserved serine or threonine residue found in the consensus sequence C2-X(4,5)-[S/T]-C3 of EGF domains, where C2 and C3 are the second and third conserved cysteines. Specifically uses GDP-fucose as donor substrate and proper disulfide pairing of the substrate EGF domains is required for fucose transfer. Plays a crucial role in NOTCH signaling. Initial fucosylation of NOTCH by POFUT1 generates a substrate for FRINGE

LOCALIZAÇÃO

Endoplasmic reticulum

VIAS BIOLÓGICAS (1)
Pre-NOTCH Processing in the Endoplasmic Reticulum
MECANISMO DE DOENÇA

Dowling-Degos disease 2

An autosomal dominant genodermatosis. Affected individuals develop a postpubertal reticulate hyperpigmentation that is progressive and disfiguring, and small hyperkeratotic dark brown papules that affect mainly the flexures and great skin folds. Patients usually show no abnormalities of the hair or nails.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
54.3 TPM
Cervix Endocervix
27.8 TPM
Aorta
27.8 TPM
Ovário
27.2 TPM
Útero
26.9 TPM
OUTRAS DOENÇAS (2)
Dowling-Degos disease 2Dowling-Degos disease
HGNC:14988UniProt:Q9H488
POMGNT2Protein O-linked-mannose beta-1,4-N-acetylglucosaminyltransferase 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

O-linked mannose beta-1,4-N-acetylglucosaminyltransferase that transfers UDP-N-acetyl-D-glucosamine to the 4-position of the mannose to generate N-acetyl-D-glucosamine-beta-1,4-O-D-mannosylprotein. Involved in the biosynthesis of the phosphorylated O-mannosyl trisaccharide (N-acetylgalactosamine-beta-3-N-acetylglucosamine-beta-4-(phosphate-6-)mannose), a carbohydrate structure present in alpha-dystroglycan (DAG1), which is required for binding laminin G-like domain-containing extracellular prote

LOCALIZAÇÃO

Endoplasmic reticulum membrane

VIAS BIOLÓGICAS (1)
DAG1 core M3 glycosylations
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A8

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with cobblestone lissencephaly and other brain anomalies, eye malformations, profound intellectual disability, and death usually in the first years of life. Included diseases are the more severe Walker-Warburg syndrome and the slightly less severe muscle-eye-brain disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Útero
98.5 TPM
Cerebelo
94.9 TPM
Cérebro - Hemisfério cerebelar
94.0 TPM
Ovário
60.0 TPM
Fallopian Tube
58.8 TPM
OUTRAS DOENÇAS (3)
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type a, 8muscular dystrophy-dystroglycanopathy (limb-girdle), type C, 8muscular dystrophy-dystroglycanopathy, type A
HGNC:25902UniProt:Q8NAT1
LFNGBeta-1,3-N-acetylglucosaminyltransferase lunatic fringeDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Glycosyltransferase that initiates the elongation of O-linked fucose residues attached to EGF-like repeats in the extracellular domain of Notch molecules. Modulates NOTCH1 activity by modifying O-fucose residues at specific EGF-like domains resulting in inhibition of NOTCH1 activation by JAG1 and enhancement of NOTCH1 activation by DLL1 via an increase in its binding to DLL1 (By similarity). Decreases the binding of JAG1 to NOTCH2 but not that of DLL1 (PubMed:11346656). Essential mediator of som

LOCALIZAÇÃO

Golgi apparatusGolgi apparatus membrane

VIAS BIOLÓGICAS (3)
Pre-NOTCH Processing in GolgiNephron developmentSomitogenesis
MECANISMO DE DOENÇA

Spondylocostal dysostosis 3, autosomal recessive

A condition of variable severity associated with vertebral and rib segmentation defects. The main skeletal malformations include fusion of vertebrae, hemivertebrae, fusion of certain ribs, and other rib malformations. Deformity of the chest and spine (severe scoliosis, kyphoscoliosis and lordosis) is a natural consequence of the malformation and leads to a dwarf-like appearance. As the thorax is small, infants frequently have respiratory insufficiency and repeated respiratory infections resulting in life-threatening complications in the first year of life.

EXPRESSÃO TECIDUAL(Ubíquo)
Skin Not Sun Exposed Suprapubic
44.0 TPM
Skin Sun Exposed Lower leg
42.0 TPM
Pâncreas
31.1 TPM
Glândula salivar
25.3 TPM
Baço
24.0 TPM
OUTRAS DOENÇAS (2)
spondylocostal dysostosis 3, autosomal recessiveautosomal recessive spondylocostal dysostosis
HGNC:6560UniProt:Q8NES3
DLL3Delta-like protein 3Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Inhibits primary neurogenesis. May be required to divert neurons along a specific differentiation pathway. Plays a role in the formation of somite boundaries during segmentation of the paraxial mesoderm (By similarity)

LOCALIZAÇÃO

Membrane

VIAS BIOLÓGICAS (2)
SomitogenesisFormation of paraxial mesoderm
MECANISMO DE DOENÇA

Spondylocostal dysostosis 1, autosomal recessive

A condition of variable severity associated with vertebral and rib segmentation defects. The main skeletal malformations include fusion of vertebrae, hemivertebrae, fusion of certain ribs, and other rib malformations. Deformity of the chest and spine (severe scoliosis, kyphoscoliosis and lordosis) is a natural consequence of the malformation and leads to a dwarf-like appearance. As the thorax is small, infants frequently have respiratory insufficiency and repeated respiratory infections resulting in life-threatening complications in the first year of life.

EXPRESSÃO TECIDUAL(Tecido-específico)
Brain Nucleus accumbens basal ganglia
11.4 TPM
Hipotálamo
8.6 TPM
Cérebro - Amígdala
8.3 TPM
Brain Anterior cingulate cortex BA24
6.8 TPM
Brain Caudate basal ganglia
6.5 TPM
OUTRAS DOENÇAS (2)
spondylocostal dysostosis 1, autosomal recessiveautosomal recessive spondylocostal dysostosis
HGNC:2909UniProt:Q9NYJ7
HES7Transcription factor HES-7Disease-causing germline mutation(s) inModerado
FUNÇÃO

Transcriptional repressor. Represses transcription from both N box- and E box-containing promoters. May with HES1, cooperatively regulate somite formation in the presomitic mesoderm (PSM). May function as a segmentation clock, which is essential for coordinated somite segmentation (By similarity)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Somitogenesis
MECANISMO DE DOENÇA

Spondylocostal dysostosis 4, autosomal recessive

A rare condition of variable severity characterized by vertebral and costal anomalies. The main feature include dwarfism, vertebral fusion, hemivertebrae, posterior rib fusion, reduced rib number, and other rib malformations.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Cerebelo
7.0 TPM
Cérebro - Hemisfério cerebelar
5.8 TPM
Linfócitos
3.0 TPM
Nervo tibial
2.0 TPM
Córtex cerebral
1.5 TPM
OUTRAS DOENÇAS (2)
spondylocostal dysostosis 4, autosomal recessiveautosomal recessive spondylocostal dysostosis
HGNC:15977UniProt:Q9BYE0
POMT1Protein O-mannosyl-transferase 1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Transfers mannosyl residues to the hydroxyl group of serine or threonine residues. Coexpression of both POMT1 and POMT2 is necessary for enzyme activity, expression of either POMT1 or POMT2 alone is insufficient (PubMed:12369018, PubMed:14699049, PubMed:28512129). Essentially dedicated to O-mannosylation of alpha-DAG1 and few other proteins but not of cadherins and protocaherins (PubMed:28512129)

LOCALIZAÇÃO

Endoplasmic reticulum membrane

VIAS BIOLÓGICAS (5)
Regulation of CDH1 posttranslational processing and trafficking to plasma membraneDAG1 core M1 glycosylationsDAG1 core M3 glycosylationsDAG1 core M2 glycosylationsDefective POMT2 causes MDDGA2, MDDGB2 and MDDGC2
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with impaired intellectual development B1

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with intellectual disability and mild structural brain abnormalities.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
73.9 TPM
Cerebelo
65.0 TPM
Cérebro - Hemisfério cerebelar
61.3 TPM
Pituitária
32.9 TPM
Ovário
27.8 TPM
OUTRAS DOENÇAS (8)
autosomal recessive limb-girdle muscular dystrophy type 2Kmuscular dystrophy-dystroglycanopathy (congenital with intellectual disability), type B1muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A1muscle-eye-brain disease
HGNC:9202UniProt:Q9Y6A1
GMPPBMannose-1-phosphate guanylyltransferase catalytic subunit betaDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Catalytic subunit of the GMPPA-GMPPB mannose-1-phosphate guanylyltransferase complex (PubMed:33986552). Catalyzes the formation of GDP-mannose, an essential precursor of glycan moieties of glycoproteins and glycolipids (PubMed:33986552). Can catalyze the reverse reaction in vitro (PubMed:33986552). Together with GMPPA regulates GDP-alpha-D-mannose levels (PubMed:33986552)

LOCALIZAÇÃO

Cytoplasm

VIAS BIOLÓGICAS (1)
Synthesis of GDP-mannose
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A14

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with brain anomalies, eye malformations, and profound intellectual disability. The disorder includes a severe form designated as Walker-Warburg syndrome and a less severe phenotype known as muscle-eye-brain disease. MDDGA14 features include increased muscle tone, microcephaly, cleft palate, feeding difficulties, severe muscle weakness, sensorineural hearing loss, cerebellar hypoplasia, ataxia, and retinal dysfunction.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Ubíquo)
Pituitária
56.4 TPM
Tireoide
41.5 TPM
Linfócitos
39.6 TPM
Próstata
37.0 TPM
Glândula salivar
32.5 TPM
OUTRAS DOENÇAS (7)
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A14autosomal recessive limb-girdle muscular dystrophy type 2Tmuscular dystrophy-dystroglycanopathy (congenital with intellectual disability), type B14muscle-eye-brain disease
HGNC:22932UniProt:Q9Y5P6
POMKProtein O-mannose kinaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Protein O-mannose kinase that specifically mediates phosphorylation at the 6-position of an O-mannose of the trisaccharide (N-acetylgalactosamine (GalNAc)-beta-1,3-N-acetylglucosamine (GlcNAc)-beta-1,4-mannose) to generate phosphorylated O-mannosyl trisaccharide (N-acetylgalactosamine-beta-1,3-N-acetylglucosamine-beta-1,4-(phosphate-6-)mannose). Phosphorylated O-mannosyl trisaccharide is a carbohydrate structure present in alpha-dystroglycan (DAG1), which is required for binding laminin G-like d

LOCALIZAÇÃO

Endoplasmic reticulum membrane

VIAS BIOLÓGICAS (1)
DAG1 core M3 glycosylations
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A12

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with cobblestone lissencephaly and other brain anomalies, eye malformations, profound intellectual disability, and death usually in the first years of life. Included diseases are the more severe Walker-Warburg syndrome and the slightly less severe muscle-eye-brain disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Linfócitos
6.4 TPM
Fibroblastos
5.9 TPM
Cérebro - Hemisfério cerebelar
3.0 TPM
Cerebelo
2.7 TPM
Testículo
2.3 TPM
OUTRAS DOENÇAS (4)
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type a, 12limb-girdle muscular dystrophy due to POMK deficiencyobsolete congenital muscular dystrophy with cerebellar involvementmuscular dystrophy-dystroglycanopathy, type A
HGNC:26267UniProt:Q9H5K3
MESP2Mesoderm posterior protein 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Transcription factor with important role in somitogenesis. Defines the rostrocaudal patterning of the somite by participating in distinct Notch pathways. Also regulates the FGF signaling pathway. Specifies the rostral half of the somites. Generates rostro-caudal polarity of somites by down-regulating in the presumptive rostral domain DLL1, a Notch ligand. Participates in the segment border formation by activating in the anterior presomitic mesoderm LFNG, a negative regulator of DLL1-Notch signal

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Somitogenesis
MECANISMO DE DOENÇA

Spondylocostal dysostosis 2, autosomal recessive

A condition of variable severity associated with vertebral and rib segmentation defects. The main skeletal malformations include fusion of vertebrae, hemivertebrae, fusion of certain ribs, and other rib malformations. Deformity of the chest and spine (severe scoliosis, kyphoscoliosis and lordosis) is a natural consequence of the malformation and leads to a dwarf-like appearance. As the thorax is small, infants frequently have respiratory insufficiency and repeated respiratory infections resulting in life-threatening complications in the first year of life.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Baixa expressão)
Testículo
1.6 TPM
Brain Frontal Cortex BA9
1.0 TPM
Córtex cerebral
0.9 TPM
Brain Nucleus accumbens basal ganglia
0.9 TPM
Glândula salivar
0.8 TPM
OUTRAS DOENÇAS (2)
spondylocostal dysostosis 2, autosomal recessiveautosomal recessive spondylocostal dysostosis
HGNC:29659UniProt:Q0VG99
B3GLCTBeta-1,3-glucosyltransferaseDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Beta-1,3-glucosyltransferase involved in one of the two pathways responsible for protein O-linked fucosylation, a unique post-translational modification of cysteine-knotted proteins that regulates various biological processes. This pathway targets proteins with Thrombospondin type-1 (TSP1) repeats (TSR) in the endoplasmic reticulum. It starts with POFUT2, which attaches fucose via an O-glycosidic bond to a conserved serine or threonine residue. B3GLCT extends this modification by transferring a

LOCALIZAÇÃO

Endoplasmic reticulum membrane

VIAS BIOLÓGICAS (1)
O-glycosylation of TSR domain-containing proteins
MECANISMO DE DOENÇA

Peters-plus syndrome

An autosomal recessive disorder characterized by anterior eye-chamber abnormalities, disproportionate short stature, developmental delay, characteristic craniofacial features, cleft lip and/or palate.

OUTRAS DOENÇAS (1)
Peters plus syndrome
HGNC:20207UniProt:Q6Y288
POMT2Protein O-mannosyl-transferase 2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Transfers mannosyl residues to the hydroxyl group of serine or threonine residues. Coexpression of both POMT1 and POMT2 is necessary for enzyme activity, expression of either POMT1 or POMT2 alone is insufficient (PubMed:14699049, PubMed:28512129). Essentially dedicated to O-mannosylation of alpha-DAG1 and few other proteins but not of cadherins and protocaherins (PubMed:28512129)

LOCALIZAÇÃO

Endoplasmic reticulum membrane

VIAS BIOLÓGICAS (5)
Regulation of CDH1 posttranslational processing and trafficking to plasma membraneDAG1 core M1 glycosylationsDAG1 core M3 glycosylationsDAG1 core M2 glycosylationsDefective POMT1 causes MDDGA1, MDDGB1 and MDDGC1
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A2

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with cobblestone lissencephaly and other brain anomalies, eye malformations, profound intellectual disability, and death usually in the first years of life. Included diseases are the more severe Walker-Warburg syndrome and the slightly less severe muscle-eye-brain disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Testículo
59.9 TPM
Pituitária
28.9 TPM
Tireoide
27.7 TPM
Cervix Endocervix
21.2 TPM
Cerebelo
21.2 TPM
OUTRAS DOENÇAS (7)
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A2muscular dystrophy-dystroglycanopathy (congenital with intellectual disability), type B2autosomal recessive limb-girdle muscular dystrophy type 2Nmuscle-eye-brain disease
HGNC:19743UniProt:Q9UKY4
FKTNRibitol-5-phosphate transferase FKTNDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Catalyzes the transfer of a ribitol-phosphate from CDP-ribitol to the distal N-acetylgalactosamine of the phosphorylated O-mannosyl trisaccharide (N-acetylgalactosamine-beta-3-N-acetylglucosamine-beta-4-(phosphate-6-)mannose), a carbohydrate structure present in alpha-dystroglycan (DAG1) (PubMed:26923585, PubMed:27194101, PubMed:29477842). This constitutes the first step in the formation of the ribitol 5-phosphate tandem repeat which links the phosphorylated O-mannosyl trisaccharide to the ligan

LOCALIZAÇÃO

Golgi apparatus membraneCytoplasmNucleus

VIAS BIOLÓGICAS (1)
Matriglycan biosynthesis on DAG1
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A4

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with cobblestone lissencephaly and other brain anomalies, eye malformations, profound intellectual disability, and death usually in the first years of life. Included diseases are the more severe Walker-Warburg syndrome and the slightly less severe muscle-eye-brain disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
21.9 TPM
Nervo tibial
12.9 TPM
Ovário
12.1 TPM
Útero
10.6 TPM
Cérebro - Hemisfério cerebelar
10.4 TPM
OUTRAS DOENÇAS (8)
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A, 4autosomal recessive limb-girdle muscular dystrophy type 2Mmuscular dystrophy-dystroglycanopathy (congenital without intellectual disability), type B4dilated cardiomyopathy 1X
HGNC:3622UniProt:O75072
RIPPLY2Protein ripply2Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Plays a role in somitogenesis. Required for somite segregation and establishment of rostrocaudal polarity in somites (By similarity)

LOCALIZAÇÃO

Nucleus

VIAS BIOLÓGICAS (1)
Somitogenesis
MECANISMO DE DOENÇA

Spondylocostal dysostosis 6, autosomal recessive

A form of spondylocostal dysostosis, a condition of variable severity associated with vertebral and rib segmentation defects. The main skeletal malformations include fusion of vertebrae, hemivertebrae, fusion of certain ribs, and other rib malformations. Deformity of the chest and spine (severe scoliosis, kyphoscoliosis and lordosis) is a natural consequence of the malformation and leads to a dwarf-like appearance. As the thorax is small, infants frequently have respiratory insufficiency and repeated respiratory infections resulting in life-threatening complications in the first year of life.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Cérebro - Hemisfério cerebelar
38.3 TPM
Cerebelo
29.7 TPM
Brain Frontal Cortex BA9
17.6 TPM
Brain Nucleus accumbens basal ganglia
16.2 TPM
Pituitária
12.6 TPM
OUTRAS DOENÇAS (2)
spondylocostal dysostosis 6, autosomal recessiveautosomal recessive spondylocostal dysostosis
HGNC:21390UniProt:Q5TAB7
FKRPRibitol 5-phosphate transferase FKRPDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Catalyzes the transfer of a ribitol 5-phosphate from CDP-L-ribitol to the ribitol 5-phosphate previously attached by FKTN/fukutin to the phosphorylated O-mannosyl trisaccharide (N-acetylgalactosamine-beta-3-N-acetylglucosamine-beta-4-(phosphate-6-)mannose), a carbohydrate structure present in alpha-dystroglycan (DAG1) (PubMed:26923585, PubMed:27194101, PubMed:29477842, PubMed:31949166). This constitutes the second step in the formation of the ribose 5-phosphate tandem repeat which links the phos

LOCALIZAÇÃO

Golgi apparatus membraneSecretedCell membrane, sarcolemmaRough endoplasmic reticulumCytoplasm

VIAS BIOLÓGICAS (1)
Matriglycan biosynthesis on DAG1
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A5

An autosomal recessive disorder characterized by congenital muscular dystrophy associated with cobblestone lissencephaly and other brain anomalies, eye malformations, profound intellectual disability, and death usually in the first years of life. Included diseases are the more severe Walker-Warburg syndrome and the slightly less severe muscle-eye-brain disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Pituitária
19.7 TPM
Útero
18.0 TPM
Fibroblastos
17.7 TPM
Tireoide
16.6 TPM
Fallopian Tube
16.5 TPM
OUTRAS DOENÇAS (8)
muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A5muscular dystrophy-dystroglycanopathy type B5autosomal recessive limb-girdle muscular dystrophy type 2Imuscle-eye-brain disease
HGNC:17997UniProt:Q9H9S5
POMGNT1Protein O-linked-mannose beta-1,2-N-acetylglucosaminyltransferase 1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Participates in O-mannosyl glycosylation by catalyzing the addition of N-acetylglucosamine to O-linked mannose on glycoproteins (PubMed:11709191, PubMed:27493216, PubMed:28512129). Catalyzes the synthesis of the GlcNAc(beta1-2)Man(alpha1-)O-Ser/Thr moiety on alpha-dystroglycan and other O-mannosylated proteins, providing the necessary basis for the addition of further carbohydrate moieties (PubMed:11709191, PubMed:27493216). Is specific for alpha linked terminal mannose and does not have MGAT3,

LOCALIZAÇÃO

Golgi apparatus membrane

VIAS BIOLÓGICAS (3)
DAG1 core M1 glycosylationsDAG1 core M2 glycosylationsMatriglycan biosynthesis on DAG1
MECANISMO DE DOENÇA

Muscular dystrophy-dystroglycanopathy congenital with brain and eye anomalies A3

An autosomal recessive disorder characterized by congenital muscular dystrophy, ocular abnormalities, cobblestone lissencephaly, and cerebellar and pontine hypoplasia. Patients present severe congenital myopia, congenital glaucoma, pallor of the optic disks, retinal hypoplasia, intellectual disability, hydrocephalus, abnormal electroencephalograms, generalized muscle weakness and myoclonic jerks. Included diseases are the more severe Walker-Warburg syndrome and the slightly less severe muscle-eye-brain disease.

EXPRESSÃO TECIDUAL(Ubíquo)
Pituitária
61.9 TPM
Tireoide
61.2 TPM
Brain Spinal cord cervical c-1
57.1 TPM
Testículo
54.0 TPM
Nervo tibial
52.7 TPM
OUTRAS DOENÇAS (8)
muscular dystrophy-dystroglycanopathy (congenital with intellectual disability), type B3muscular dystrophy-dystroglycanopathy (congenital with brain and eye anomalies), type A3retinitis pigmentosa 76autosomal recessive limb-girdle muscular dystrophy type 2O
HGNC:19139UniProt:Q8WZA1

Variantes genéticas (ClinVar)

564 variantes patogênicas registradas no ClinVar.

🧬 POGLUT1: NM_152305.3(POGLUT1):c.457-2A>G ()
🧬 POGLUT1: NM_152305.3(POGLUT1):c.574dup (p.Val192fs) ()
🧬 POGLUT1: NM_152305.3(POGLUT1):c.*132A>T ()
🧬 POGLUT1: NM_152305.3(POGLUT1):c.966-13C>G ()
🧬 POGLUT1: NM_152305.3(POGLUT1):c.1169_*17del (p.Thr390fs) ()
Ver todas no ClinVar

Vias biológicas (Reactome)

38 vias biológicas associadas aos genes desta condição.

Pre-NOTCH Processing in the Endoplasmic Reticulum Formation of paraxial mesoderm Somitogenesis Formation of the posterior neural plate Matriglycan biosynthesis on DAG1 Type I hemidesmosome assembly Keratinization Formation of the cornified envelope Differentiation of Keratinocytes in Interfollicular Epidermis in Mammalian Skin Developmental Lineage of Mammary Gland Luminal Epithelial Cells Developmental Lineage of Mammary Gland Myoepithelial Cells Developmental Lineage of Mammary Stem Cells Nuclear signaling by ERBB4 Regulated proteolysis of p75NTR NRIF signals cell death from the nucleus Activated NOTCH1 Transmits Signal to the Nucleus Constitutive Signaling by NOTCH1 PEST Domain Mutants Constitutive Signaling by NOTCH1 HD+PEST Domain Mutants NOTCH2 Activation and Transmission of Signal to the Nucleus EPH-ephrin mediated repulsion of cells NOTCH3 Activation and Transmission of Signal to the Nucleus NOTCH4 Activation and Transmission of Signal to the Nucleus Noncanonical activation of NOTCH3 Amyloid fiber formation TGFBR3 PTM regulation DAG1 core M3 glycosylations Pre-NOTCH Processing in Golgi Defective LFNG causes SCDO3 Nephron development Defective POMT2 causes MDDGA2, MDDGB2 and MDDGC2 Defective POMT1 causes MDDGA1, MDDGB1 and MDDGC1 DAG1 core M2 glycosylations DAG1 core M1 glycosylations Regulation of CDH1 posttranslational processing and trafficking to plasma membrane Synthesis of GDP-mannose Defective B3GALTL causes PpS O-glycosylation of TSR domain-containing proteins Defective POMGNT1 causes MDDGA3, MDDGB3 and MDDGC3

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Onde tratar no SUS

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🇧🇷 Atendimento SUS — Alteração da proteína O-glicosilação

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Publicações mais relevantes

Timeline de publicações
0 papers (10 anos)
#1

GALNT3 Inhibits the Progression of Cerebral Ischemia-Reperfusion Injury by Stabilizing TREM2 via O-GalNAc Glycosylation.

CNS neuroscience & therapeutics2026 Mar

A lesão por isquemia-reperfusão cerebral (LIRC) após um AVC é um desafio sem terapias eficazes. Este estudo demonstrou que a enzima GALNT3, cuja expressão diminui na LIRC, pode reduzir significativamente o volume do infarto e melhorar a função neurológica em modelos animais, ao diminuir a inflamação e a morte celular. Isso ocorre porque a GALNT3 estabiliza a proteína TREM2 através de um processo de glicosilação, sugerindo que a manipulação dessa via pode ser uma nova estratégia terapêutica promissora para proteger o cérebro de pacientes com AVC.

🇧🇷 traduzido
#2

GalNAc-T13 maintains neurite architecture and memory retention via O-GalNAc glycosylation of seizure protein 6.

Proceedings of the National Academy of Sciences of the United States of America2026 Mar 10

Este estudo demonstra que a enzima GalNAc-T13 é crucial para a formação e manutenção da arquitetura dos neurônios no cérebro (neurite e ramificação dendrítica) e para a consolidação da memória. Sua disfunção, observada em modelos de camundongos, leva a problemas na estrutura cerebral e déficits de memória, que em pacientes poderiam se manifestar como dificuldades cognitivas ou atrasos no desenvolvimento. A GalNAc-T13 age modificando (glicosilando) a proteína SEZ6, processo essencial para a estabilidade e função desta no crescimento neuronal. Compreender este mecanismo oferece insights importantes para médicos e pesquisadores sobre a base molecular de certos distúrbios neuropsiquiátricos e aponta para possíveis alvos diagnósticos e terapêuticos futuros.

🇧🇷 traduzido
#3

Probing APP Cleavage and Amyloid-β Assembly via Synthetic MUC-Type O-Glycosylated APP Glycopeptides.

ACS chemical neuroscience2026 Mar 18

A glicosilação (adição de açúcares) na proteína precursora amiloide (APP) desempenha um papel fundamental na doença de Alzheimer, influenciando como a APP é processada e como os peptídeos amiloides tóxicos se agregam no cérebro. Este estudo revela que a localização e a quantidade desses açúcares O-GalNAc na APP podem tanto promover a formação de agregados amiloides prejudiciais (especialmente em mutações ligadas à doença), quanto, em outras configurações, desorganizá-los. Compreender esses mecanismos oferece novas perspectivas para o desenvolvimento de diagnósticos e terapias focadas nessas modificações de açúcar para combater a doença.

🇧🇷 traduzido
#4

Interferon-stimulated gene GALNT2 restricts respiratory virus infections.

Nature microbiology2026 Jan

Este estudo identifica o gene GALNT2, responsável pela O-glicosilação de proteínas, como um importante fator antiviral da imunidade inata. Ele restringe a replicação de vírus respiratórios como coronavírus e influenza, contribuindo para a redução da carga viral e da gravidade da doença. Para pacientes e médicos, é crucial saber que variantes genéticas do GALNT2 com perda de função antiviral foram associadas a um risco elevado de hospitalização após infecção por SARS-CoV-2, destacando a importância da função de O-glicosilação mediada por GALNT2 na defesa contra essas infecções.

🇧🇷 traduzido
#5

Disrupted O-GalNAc glycosylation as a mechanism and biomarker of SLC35A2 -associated epilepsy.

bioRxiv : the preprint server for biology2026 Mar 04

Este estudo revela que a epilepsia grave e os distúrbios neurológicos causados por alterações genéticas no gene SLC35A2 resultam de um defeito específico na produção de um tipo de cadeia de açúcar, as glicanas O-GalNAc, essencial para o desenvolvimento e funcionamento dos neurônios. Essa falha na formação desses açúcares compromete a função neuronal, levando à sua hiperexcitabilidade, o que foi confirmado em modelos e em tecidos cerebrais de pacientes. Para pacientes e médicos, essa descoberta é crucial porque estabelece um mecanismo claro para a doença, abrindo caminho para o desenvolvimento de novos biomarcadores para diagnóstico e monitoramento, e terapias direcionadas para a epilepsia associada ao SLC35A2.

🇧🇷 traduzido

Publicações recentes

Ver todas no PubMed

📚 EuropePMCmostrando 199

2026

Disrupted O-GalNAc glycosylation as a mechanism and biomarker of SLC35A2 -associated epilepsy.

bioRxiv : the preprint server for biology
2026

Fine structural features of polysaccharides and gut microbiota Co-regulate mucin O-glycosylation: Mechanisms and advances.

Carbohydrate polymers
2026

GALNT3 Inhibits the Progression of Cerebral Ischemia-Reperfusion Injury by Stabilizing TREM2 via O-GalNAc Glycosylation.

CNS neuroscience & therapeutics
2026

GalNAc-T13 maintains neurite architecture and memory retention via O-GalNAc glycosylation of seizure protein 6.

Proceedings of the National Academy of Sciences of the United States of America
2026

Glycoprotein biomarkers for bladder cancer detection.

Clinica chimica acta; international journal of clinical chemistry
2026

Probing APP Cleavage and Amyloid-β Assembly via Synthetic MUC-Type O-Glycosylated APP Glycopeptides.

ACS chemical neuroscience
2026

Epigenetic and O-glycosylation regulation of p66Shc mitigates mitochondrial oxidative stress in aortic dissection.

Theranostics
2026

Clinical, biochemical and genetic characterization of an Egyptian patient with SRD5A3-congenital glycosylation disorder.

Ophthalmic genetics
2026

Multi-omics analyses reveal the pathogenic role of terminal ileum-derived IgA+β7+ cells in IgA nephropathy.

Kidney international
2026

Emergence and epidemiology of dominant variants of human metapneumovirus in the United States between 2016 and 2021.

mBio
2025

Unlocking the Sugar Code: Implications and Consequences of Glycosylation in Alzheimer's Disease and Other Tauopathies.

Biomedicines
2025

GALNT7 promotes hepatocellular carcinoma progression by activating the PI3K/AKT signaling pathway via O-glycosylation of MUC13.

Acta biochimica et biophysica Sinica
2025

Mucin-type O-glycans regulate proteoglycan stability and chondrocyte maturation.

bioRxiv : the preprint server for biology
2025

Dynamic glycosylation remodeling in neurological disorders.

Frontiers in molecular neuroscience
2025

GALNT3 is a novel target driving lymphomagenesis via O-glycosylation of FGFR2.

Cell communication and signaling : CCS
2026

Interferon-stimulated gene GALNT2 restricts respiratory virus infections.

Nature microbiology
2025

Comparative immunoinformatic analysis of Rhipicephalus microplus cocktail vaccine targets.

Parasites & vectors
2025

Insights Into the Pathological Glycosylation Associated With COG6-CDG.

Human mutation
2026

α-O-Glycosylation at Tyrosine 10 Promotes the Astrocyte Clearance of Amyloid-β Peptide 1-42.

Chembiochem : a European journal of chemical biology
2025

Decoding the complex substrate specificities of GalNAc-Ts.

Glycobiology
2025

In vitro cell model to dilucidate the underlying molecular mechanism associated with ophthalmic manifestation of congenital disorders of glycosylation: studying an ALG2-CDG patient.

Frontiers in genetics
2025

Potential Function of Glycosylated RNA in Diseases.

Wiley interdisciplinary reviews. RNA
2025

Glycan Signatures on Neutrophils in an Equine Model for Autoimmune Uveitis.

Biomolecules
2025

ATP6AP2-Related Disease Caused by Splicing Defects: Abnormal Glycosylation and the First Affected Female.

Journal of inherited metabolic disease
2025

Molecular dynamics study of the impact of glycosylation on conformational properties of trimeric N-terminal domain of human copper transporter 1.

Carbohydrate research
2025

The Importance of N- and O-Glycosylation of Brain Cell Surface Glycoproteins.

Glycobiology
2025

MIC13 of Toxoplasma gondii: Potential Gene for Vaccine Candidate-An In Silico Approach.

Journal of parasitology research
2025

Abnormal O-glycan sialylation in the mPFC contributes to depressive-like behaviors in male mice.

Science advances
2025

Neuromuscular Defects in a Drosophila Model of the Congenital Disorder of Glycosylation SLC35A2-CDG.

Biomolecules
2026

Salivary Nitrate Maintains Mucosal Homeostasis via the Sialin-Neuropeptide Axis.

Journal of dental research
2025

The β-1,4 GalT-V Interactome-Potential Therapeutic Targets and a Network of Pathways Driving Cancer and Cardiovascular and Inflammatory Diseases.

International journal of molecular sciences
2025

Protein glycosylation modification as a novel forensic biomarker for discriminating monozygotic twins.

Forensic science international
2025

Hylocereus polyrhizus Pulp Residues Polysaccharide Alleviates High-Fat Diet-Induced Obesity by Modulating Intestinal Mucus Secretion and Glycosylation.

Foods (Basel, Switzerland)
2025

Is dolichol pathway dysfunction a significant factor in Alzheimer's disease?

Inflammopharmacology
2025

Integrated multi-omics analysis and machine learning refine molecular subtypes and prognosis in hepatocellular carcinoma through O-linked glycosylation genes.

Functional & integrative genomics
2025

Approaches to diagnostic screening for congenital disorders of glycosylation and its prevalence in Japan.

Journal of human genetics
2025

GlycoIP: an integrated platform for simultaneous and site-specific N/O-glycosylation analysis of human semen.

Frontiers in chemistry
2025

Map of the neuronal O-glycoproteome reveals driver functions in the regulated secretory pathway.

The Journal of biological chemistry
2025

GALNT14 deficiency: connecting multiple links in the IgA nephropathy pathogenetic chain.

The Journal of clinical investigation
2025

Implications of Mucin-Type O-Glycosylation in Alzheimer's Disease.

Molecules (Basel, Switzerland)
2025

Molecular mechanisms and pathophysiological implications of mucin-type O-glycosylation dysregulation in colorectal cancer progression.

Naunyn-Schmiedeberg's archives of pharmacology
2025

Myriad mechanisms: factors regulating the synthesis of aberrant mucin-type O-glycosylation found on cancer cells.

Glycobiology
2025

New insights into phytochemicals via protein glycosylation focused on aging and diabetes.

Phytomedicine : international journal of phytotherapy and phytopharmacology
2025

Glycosylation in Dermatology: Unveiling the Sugar Coating of Skin Disease.

Experimental dermatology
2025

Clinical and Molecular Features of Patients With Congenital Disorders of Glycosylation in Japan.

JIMD reports
2025

Loss of GalNAc-T14 links O-glycosylation defects to alterations in B cell homing in IgA nephropathy.

The Journal of clinical investigation
2025

Diagnostic and Therapeutic Approaches in Congenital Disorders of Glycosylation.

Handbook of experimental pharmacology
2025

SPOP/NOLC1/B4GALT1 signaling axis enhances paclitaxel resistance in endometrial cancer by inducing O-dysglycosylation.

Oncogene
2025

Exploring the combined roles of GALNT1 and GALNT2 in hepatocellular carcinoma malignancy and EGFR modulation.

Discover oncology
2025

ER O-glycosylation in synovial fibroblasts drives cartilage degradation.

Nature communications
2024

Emerging Biochemical and Immunologic Mechanisms in the Pathogenesis of IgA Nephropathy.

Seminars in nephrology
2025

RCL glycosylation of serum corticosteroid-binding globulin: implications in cortisol delivery and septic shock.

Glycobiology
2025

Antibodies with specificity to glycan motifs that decorate OMV cargo proteins.

mSphere
2025

O-GalNAc glycans are enriched in neuronal tracts and regulate nodes of Ranvier.

Proceedings of the National Academy of Sciences of the United States of America
2025

C1GALT1C1-Associated Mosaic Disorder of Glycosylation in a Female.

Journal of inherited metabolic disease
2025

Mass Spectrometry as a First-Line Diagnostic Aid for Congenital Disorders of Glycosylation.

Mass spectrometry (Tokyo, Japan)
2025

Exonic CircGUCY1A2 inhibits pulmonary artery smooth muscle cells phenotypic switching via regulating O-glycosylation of COL3A1 in pulmonary hypertension.

European journal of pharmacology
2025

O-GlcNAc modification differentially regulates microtubule binding and pathological conformations of tau isoforms in vitro.

The Journal of biological chemistry
2025

Enhanced O-glycosylation site prediction using explainable machine learning technique with spatial local environment.

Bioinformatics (Oxford, England)
2025

Brain microenvironment-remodeling nanomedicine improves cerebral glucose metabolism, mitochondrial activity and synaptic function in a mouse model of Alzheimer's disease.

Biomaterials
2025

Glycosylation Pathways Targeted by Deregulated miRNAs in Autism Spectrum Disorder.

International journal of molecular sciences
2025

Decoding Extracellular Protein Glycosylation in Human Health and Disease.

Annual review of analytical chemistry (Palo Alto, Calif.)
2025

SLC10A7 regulates O-GalNAc glycosylation and Ca2+ homeostasis in the secretory pathway: insights into SLC10A7-CDG.

Cellular and molecular life sciences : CMLS
2024

Golgi pH elevation due to loss of V-ATPase subunit V0a2 function correlates with tissue-specific glycosylation changes and globozoospermia.

Cellular and molecular life sciences : CMLS
2025

"Intrinsic disorder-protein modification-LLPS-tumor" regulatory axis: From regulatory mechanisms to precision medicine.

Biochimica et biophysica acta. Reviews on cancer
2024

GalNT2-mediated O-glycosylation affects pancreas development and function in mice.

Scientific reports
2025

GALNT6, transcriptionally inhibited by KLF9, promotes osteosarcoma progression by increasing EFEMP1 expression via O-glycosylation modification.

Biochimica et biophysica acta. Molecular cell research
2025

C1GALT1 high expression enhances the progression of glioblastoma through the EGFR-AKT/ERK cascade.

Cellular signalling
2025

GALNT3 in Ischemia-Reperfusion Injury of the Kidney.

Journal of the American Society of Nephrology : JASN
2024

MALAT1/miR-582-5p/GALNT1/MUC1 axis modulates progression of AML leukemia stem cells by regulating JAK2/STAT3 pathway.

Annals of hematology
2024

Giantin mediates Golgi localization of Gal3-O-sulfotransferases and affects salivary mucin sulfation in patients with Sjögren's disease.

JCI insight
2024

Rapid identification of primary atopic disorders (PAD) by a clinical landmark-guided, upfront use of genomic sequencing.

Allergologie select
2024

Hormonal and Allosteric Regulation of the Luteinizing Hormone/Chorionic Gonadotropin Receptor.

Frontiers in bioscience (Landmark edition)
2024

Aberrant glycosylation in schizophrenia: insights into pathophysiological mechanisms and therapeutic potentials.

Frontiers in pharmacology
2024

O-Glycosylation of a male seminal fluid protein influences sperm binding and female postmating behavior.

PNAS nexus
2024

Clinical glycoprotein mass spectrometry: The future of disease detection and monitoring.

Journal of mass spectrometry : JMS
2024

The genetics and epidemiology of N- and O-immunoglobulin A glycomics.

Genome medicine
2024

In vivo mapping of the mouse Galnt3-specific O-glycoproteome.

The Journal of biological chemistry
2024

O-glycosylation of IgA1 and the pathogenesis of an autoimmune disease IgA nephropathy.

Glycobiology
2024

Site-Specific Glycosylation Analysis of Human and Murine Fcγ Receptor II Family Members Reveals Variant-Specific N-Glycosylation.

Journal of proteome research
2024

B3galt5 functions as a PXR target gene and regulates obesity and insulin resistance by maintaining intestinal integrity.

Nature communications
2024

Insights into molecular and cellular functions of the Golgi calcium/manganese-proton antiporter TMEM165.

The Journal of biological chemistry
2024

Computational design of novel chimeric multiepitope vaccine against bacterial and viral disease in tilapia (Oreochromis sp.).

Scientific reports
2024

B3GNT7 regulates mucin O-glycosylation to alleviate colonic inflammation.

BMC gastroenterology
2024

The intriguing strategies of Tannerella forsythia's host interaction.

Frontiers in oral health
2024

Apolipoprotein-CIII O-Glycosylation Is Associated with Micro- and Macrovascular Complications of Type 2 Diabetes.

International journal of molecular sciences
2024

Matriglycan maintains t-tubule structural integrity in cardiac muscle.

Proceedings of the National Academy of Sciences of the United States of America
2024

Diaminocyclopentane - l-Lysine Adducts: Potent and selective inhibitors of human O-GlcNAcase.

Bioorganic chemistry
2024

Biochemical diagnosis of congenital disorders of glycosylation.

Advances in clinical chemistry
2024

Comprehensive Glycosylation Characterization of Recombinant Human Erythropoietin by Electron-Activated Dissociation Mass Spectrometry.

Applied biochemistry and biotechnology
2024

A Toxoplasma gondii O-glycosyltransferase that modulates bradyzoite cyst wall rigidity is distinct from host homologues.

Nature communications
2024

The importance of muscle glycogen phosphorylase in glial cells function.

Biochemical Society transactions
2024

Genomic analysis of canine pneumoviruses and canine respiratory coronavirus from New Zealand.

New Zealand veterinary journal
2024

O-glycan determinants regulate VWF trafficking to Weibel-Palade bodies.

Blood advances
2024

Stool Glycoproteomics Signatures of Pre-Cancerous Lesions and Colorectal Cancer.

International journal of molecular sciences
2024

Impaired Glycosylation of Gastric Mucins Drives Gastric Tumorigenesis and Serves as a Novel Therapeutic Target.

Gastroenterology
2024

Protein glycosylation in lung cancer from a mass spectrometry perspective.

Mass spectrometry reviews
2024

Profiling Human Cerebrospinal Fluid (CSF) Endogenous Peptidome in Alzheimer's Disease.

Methods in molecular biology (Clifton, N.J.)
2024

Aberrantly Glycosylated GLUT1 as a Poor Prognosis Marker in Aggressive Bladder Cancer.

International journal of molecular sciences
2024

Ion mobility-tandem mass spectrometry of mucin-type O-glycans.

Nature communications
2024

Emerging Roles of UDP-GalNAc Polypeptide N-Acetylgalactosaminyltransferases in Cardiovascular Disease.

Aging and disease
2024

Functional prediction of proteins from the human gut archaeome.

ISME communications
2024

Loss of the glycosyltransferase Galnt11 affects vitamin D homeostasis and bone composition.

The Journal of biological chemistry
2024

Examining the association between serum galactose-deficient IgA1 and primary IgA nephropathy: a systematic review and meta-analysis.

Journal of nephrology
2024

Deficiency of polypeptide N-acetylgalactosamine transferase 9 contributes to a risk for Parkinson's disease via mitochondrial dysfunctions.

International journal of biological macromolecules
2025

HSF1 Increases EOGT-Mediated Glycosylation of Notch1 to Promote IL-1β-Induced Inflammatory Injury of Chondrocytes.

Cartilage
2024

Characterization of MdpS: an in-depth analysis of a MUC5B-degrading protease from Streptococcus oralis.

Frontiers in microbiology
2024

O-glycoprofiling of Serum Apolipoprotein C-III in Colorectal Cancer.

Frontiers in bioscience (Landmark edition)
2024

Fecal-adherent mucus is a non-invasive source of primary human MUC2 for structural and functional characterization in health and disease.

The Journal of biological chemistry
2024

TRIM14 suppressed the progression of NSCLC via hexosamine biosynthesis pathway.

Carcinogenesis
2024

Cancer snap-shots: Biochemistry and glycopathology of O-glycans: A review.

International journal of biological macromolecules
2024

Protein O-GlcNAcylation: The sweet hub in liver metabolic flexibility from a (patho)physiological perspective.

Liver international : official journal of the International Association for the Study of the Liver
2023

Advances in Bacterial Oligosaccharyltransferase Structure Elucidation and Potential Application to Glycoconjugate Vaccine Design.

Frontiers in bioscience (Landmark edition)
2024

H3K27 acetylation activated-PDLIM7 promotes castration-resistant prostate cancer progression by inducing O-Glycosylation of YAP1 protein.

Translational oncology
2023

Talniflumate abrogates mucin immune suppressive barrier improving efficacy of gemcitabine and nab-paclitaxel treatment in pancreatic cancer.

Journal of translational medicine
2024

Impaired digestive function of sucrase-isomaltase in a complex with the Greenlandic sucrase-isomaltase variant.

Biochimica et biophysica acta. Molecular basis of disease
2023

GlycoTCFM: Glycoproteomics Based on Two Complementary Fragmentation Methods Reveals Distinctive O-Glycosylation in Human Sperm and Seminal Plasma.

Journal of proteome research
2023

Muc2 mucin O-glycosylation interacts with enteropathogenic Escherichia coli to influence the development of ulcerative colitis based on the NF-kB signaling pathway.

Journal of translational medicine
2023

Quantitative mapping of the in vivo O-GalNAc glycoproteome in mouse tissues identifies GalNAc-T2 O-glycosites in metabolic disorder.

Proceedings of the National Academy of Sciences of the United States of America
2023

Apolipoprotein-CIII O-Glycosylation, a Link between GALNT2 and Plasma Lipids.

International journal of molecular sciences
2023

Exploiting Chemical Protein Synthesis to Study the Role of Tyrosine Sulfation on Anticoagulants from Hematophagous Organisms.

Accounts of chemical research
2023

Proteomic analysis of diabetic retinas.

Frontiers in endocrinology
2023

Highly functionalized diaminocyclopentanes: A new route to potent and selective inhibitors of human O-GlcNAcase.

Bioorganic chemistry
2023

Protein glycosylation: bridging maternal-fetal crosstalk during embryo implantation†.

Biology of reproduction
2023

Polypeptide N-acetylgalactosaminyltransferase (GalNAc-T) isozyme surface charge governs charge substrate preferences to modulate mucin type O-glycosylation.

Glycobiology
2023

In Silico Characterization of an Important Metacyclogenesis Marker in Leishmania donovani, HASPB1, as a Potential Vaccine Candidate.

BioMed research international
2023

Deciphering Protein O-GalNAcylation: Method Development and Disease Implication.

ACS omega
2023

O-GalNAc glycosylation determines intracellular trafficking of APP and Aβ production.

The Journal of biological chemistry
2023

Germline C1GALT1C1 mutation causes a multisystem chaperonopathy.

Proceedings of the National Academy of Sciences of the United States of America
2023

Glycosylation and behavioral symptoms in neurological disorders.

Translational psychiatry
2023

Emerging roles of O-glycosylation in regulating protein aggregation, phase separation, and functions.

Current opinion in chemical biology
2023

Altered Glycosylation in Progression and Management of Bladder Cancer.

Molecules (Basel, Switzerland)
2023

Clinical Presentation of a Patient with a Congenital Disorder of Glycosylation, Type IIs (ATP6AP1), and Liver Transplantation.

International journal of molecular sciences
2023

Apolipoprotein E O-glycosylation is associated with amyloid plaques and APOE genotype.

Analytical biochemistry
2023

Biochemical characterization, and anti-inflammatory and antitumor activities of glycoprotein from lamb abomasum.

Journal of ethnopharmacology
2023

Indoxyl sulfate induces left ventricular hypertrophy via the AhR-FGF23-FGFR4 signaling pathway.

Frontiers in cardiovascular medicine
2023

Identification of global inhibitors of cellular glycosylation.

Nature communications
2023

In-depth quantitative proteomics analysis revealed C1GALT1 depletion in ECC-1 cells mimics an aggressive endometrial cancer phenotype observed in cancer patients with low C1GALT1 expression.

Cellular oncology (Dordrecht, Netherlands)
2022

The (Sialyl) Tn antigen: Contributions to immunosuppression in gastrointestinal cancers.

Frontiers in oncology
2022

Case report: The art of anesthesiology-Approaching a minor procedure in a child with MPI-CDG.

Frontiers in pharmacology
2022

Emerging Roles of the Unique Molecular Chaperone Cosmc in the Regulation of Health and Disease.

Biomolecules
2022

GALNT1 Enhances Malignant Phenotype of Gastric Cancer via Modulating CD44 Glycosylation to Activate the Wnt/β-catenin Signaling Pathway.

International journal of biological sciences
2023

Simultaneous Quantification of Apolipoprotein C-III O-Glycoforms by Protein-MRM.

Journal of proteome research
2022

Development of an enrichment-free one-pot sample preparation and ultra-high performance liquid chromatography-tandem mass spectrometry method to identify Immunoglobulin A1 hinge region O-glycoforms for Immunoglobulin A nephropathy.

Journal of chromatography. A
2023

A State of Natriuretic Peptide Deficiency.

Endocrine reviews
2022

Glycosylation in SARS-CoV-2 variants: A path to infection and recovery.

Biochemical pharmacology
2022

Protein O-GlcNAcylation in Metabolic Modulation of Skeletal Muscle: A Bright but Long Way to Go.

Metabolites
2022

Global mapping of GalNAc-T isoform-specificities and O-glycosylation site-occupancy in a tissue-forming human cell line.

Nature communications
2022

Clinical and molecular findings in three Japanese patients with N-acetylneuraminic acid synthetase-congenital disorder of glycosylation (NANS-CDG).

Scientific reports
2022

A novel FGF23 mutation in hyperphosphatemic familial tumoral calcinosis and its deleterious effect on protein O-glycosylation.

Frontiers in endocrinology
2022

Fringe-positive Golgi outposts unite temporal Furin 2 convertase activity and spatial Delta signal to promote dendritic branch retraction.

Cell reports
2022

Lysosomal cathepsin D mediates endogenous mucin glycodomain catabolism in mammals.

Proceedings of the National Academy of Sciences of the United States of America
2022

O-Glycosylating Enzyme GALNT2 Predicts Worse Prognosis in Cervical Cancer.

Pathology oncology research : POR
2022

Recent advances in demystifying O-glycosylation in health and disease.

Proteomics
2022

The role and potential mechanism of O-Glycosylation in gastrointestinal tumors.

Pharmacological research
2022

Heterogeneous glycosylation and methylation of the Aeromonas caviae flagellin.

MicrobiologyOpen
2022

Si-Wu Water Extracts Protect against Colonic Mucus Barrier Damage by Regulating Muc2 Mucin Expression in Mice Fed a High-Fat Diet.

Foods (Basel, Switzerland)
2022

O-Glycosylation Changes in Serum Immunoglobulin G Are Associated with Inflammation Development in Advanced Endometriosis.

International journal of molecular sciences
2022

O-Glycan-Dependent Interaction between MUC1 Glycopeptide and MY.1E12 Antibody by NMR, Molecular Dynamics and Docking Simulations.

International journal of molecular sciences
2022

Post-Translational Modifications in Atopic Dermatitis: Current Research and Clinical Relevance.

Frontiers in cell and developmental biology
2022

Advances in glycopeptide enrichment methods for the analysis of protein glycosylation over the past decade.

Journal of separation science
2022

Glycosylation of HDL-Associated Proteins and Its Implications in Cardiovascular Disease Diagnosis, Metabolism and Function.

Frontiers in cardiovascular medicine
2022

Differential Effects of D-Galactose Supplementation on Golgi Glycosylation Defects in TMEM165 Deficiency.

Frontiers in cell and developmental biology
2022

Mucin-Type O-Glycosylation Proximal to β-Secretase Cleavage Site Affects APP Processing and Aggregation Fate.

Frontiers in chemistry
2022

The barrier and beyond: Roles of intestinal mucus and mucin-type O-glycosylation in resistance and tolerance defense strategies guiding host-microbe symbiosis.

Gut microbes
2022

In-Depth Profiling of O-Glycan Isomers in Human Cells Using C18 Nanoliquid Chromatography-Mass Spectrometry and Glycogenomics.

Analytical chemistry
2022

Global Loss of Core 1-Derived O-Glycans in Mice Leads to High Mortality Due to Acute Kidney Failure and Gastric Ulcers.

International journal of molecular sciences
2022

Pan-cancer analysis of GALNTs expression identifies a prognostic of GALNTs feature in low grade glioma.

Journal of leukocyte biology
2022

COG6-CDG: Novel variants and novel malformation.

Birth defects research
2022

Characterization of the O-Glycoproteome of Porphyromonas gingivalis.

Microbiology spectrum
2022

GALNT4 primes monocytes adhesion and transmigration by regulating O-Glycosylation of PSGL-1 in atherosclerosis.

Journal of molecular and cellular cardiology
2022

In-Depth Site-Specific O-Glycosylation Analysis of Glycoproteins and Endogenous Peptides in Cerebrospinal Fluid (CSF) from Healthy Individuals, Mild Cognitive Impairment (MCI), and Alzheimer's Disease (AD) Patients.

ACS chemical biology
2021

Asparagine Tautomerization in Glycosyltransferase Catalysis. The Molecular Mechanism of Protein O-Fucosyltransferase 1.

ACS catalysis
2021

O-Glycosylation Induces Amyloid-β To Form New Fibril Polymorphs Vulnerable for Degradation.

Journal of the American Chemical Society
2021

Integrated N- and O-Glycomics of Acute Myeloid Leukemia (AML) Cell Lines.

Cells
2021

Altered Mucus Barrier Integrity and Increased Susceptibility to Colitis in Mice upon Loss of Telocyte Bone Morphogenetic Protein Signalling.

Cells
2021

Mucin-Type O-Glycans: Barrier, Microbiota, and Immune Anchors in Inflammatory Bowel Disease.

Journal of inflammation research
2021

O-GlcNAcase inhibitor has protective effects in intracerebral hemorrhage by suppressing the inflammatory response.

Neuroreport
2021

Immunoglobulin A Glycosylation and Its Role in Disease.

Experientia supplementum (2012)
2021

Lectin and Liquid Chromatography-Based Methods for Immunoglobulin (G) Glycosylation Analysis.

Experientia supplementum (2012)
2021

Polypeptide N-acetylgalactosaminyltransferase-Associated Phenotypes in Mammals.

Molecules (Basel, Switzerland)
2021

O-Glycosylation Landscapes of SARS-CoV-2 Spike Proteins.

Frontiers in chemistry
2021

Characterization of the O-Glycoproteome of Tannerella forsythia.

mSphere
2021

Mucolytic bacteria: prevalence in various pathological diseases.

World journal of microbiology & biotechnology
2021

Mucin-Type O-GalNAc Glycosylation in Health and Disease.

Advances in experimental medicine and biology
2021

A novel mechanism for C1GALT1 in the regulation of gastric cancer progression.

Cell & bioscience
2021

Global functions of O-glycosylation: promises and challenges in O-glycobiology.

The FEBS journal
2021

O-glycosylation pattern of the SARS-CoV-2 spike protein reveals an "O-Follow-N" rule.

Cell research
2021

Tyrosine O-GalNAc Alters the Conformation and Proteolytic Susceptibility of APP Model Glycopeptides.

ACS chemical neuroscience
2021

A semi-automated, high throughput approach for O-glycosylation profiling of in vitro established cancer cell lines by MALDI-FT-ICR MS.

Glycoconjugate journal
2021

Comprehensive analysis of O-glycosylation of amyloid precursor protein (APP) using targeted and multi-fragmentation MS strategy.

Biochimica et biophysica acta. General subjects
2021

The O-Glycome of Human Nigrostriatal Tissue and Its Alteration in Parkinson's Disease.

Journal of proteome research
2021

O-glycoforms of polymeric immunoglobulin A1 in the plasma of patients with IgA nephropathy are associated with pathological phenotypes.

Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association
2021

C1GALT1 in health and disease.

Oncology letters
2021

O- and N-Glycosylation of Serum Immunoglobulin A is Associated with IgA Nephropathy and Glomerular Function.

Journal of the American Society of Nephrology : JASN
2021

BAG3 epigenetically regulates GALNT10 expression via WDR5 and facilitates the stem cell-like properties of platin-resistant ovarian cancer cells.

Biochimica et biophysica acta. Molecular cell research
2021

Oxonium Ion Guided Analysis of Quantitative Proteomics Data Reveals Site-Specific O-Glycosylation of Anterior Gradient Protein 2 (AGR2).

International journal of molecular sciences
2021

Proteoglycan synthesis in conserved oligomeric Golgi subunit deficient HEK293T cells is affected differently, depending on the lacking subunit.

Traffic (Copenhagen, Denmark)
2021

Slimy partners: the mucus barrier and gut microbiome in ulcerative colitis.

Experimental & molecular medicine

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Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. GALNT3 Inhibits the Progression of Cerebral Ischemia-Reperfusion Injury by Stabilizing TREM2 via O-GalNAc Glycosylation.
    CNS neuroscience & therapeutics· 2026· PMID 41814467mais citado
  2. GalNAc-T13 maintains neurite architecture and memory retention via O-GalNAc glycosylation of seizure protein 6.
    Proceedings of the National Academy of Sciences of the United States of America· 2026· PMID 41790942mais citado
  3. Probing APP Cleavage and Amyloid-β Assembly via Synthetic MUC-Type O-Glycosylated APP Glycopeptides.
    ACS chemical neuroscience· 2026· PMID 41738447mais citado
  4. Interferon-stimulated gene GALNT2 restricts respiratory virus infections.
    Nature microbiology· 2026· PMID 41387548mais citado
  5. Disrupted O-GalNAc glycosylation as a mechanism and biomarker of SLC35A2 -associated epilepsy.
    bioRxiv : the preprint server for biology· 2026· PMID 41867720mais citado
  6. PGM1 deficiency: Substrate use during exercise and effect of treatment with galactose.
    Neuromuscul Disord· 2017· PMID 28190645recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:309447(Orphanet)
  2. MONDO:0017741(MONDO)
  3. GARD:21336(GARD (NIH))
  4. Variantes catalogadas(ClinVar)
  5. Busca completa no PubMed(PubMed)
  6. Q55787317(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

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Alteração da proteína O-glicosilação
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Alteração da proteína O-glicosilação

ORPHA:309447 · MONDO:0017741
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C5681041
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