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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
GNS
glucosamine (N-acetyl)-6-sulfatase
Chromosome 12 Β· 12q14.3
NCBI Gene: 2799Ensembl: ENSG00000135677.12HGNC: HGNC:4422UniProt: A0A024RBC5
54PubMed Papers
21Diseases
0Drugs
83Pathogenic Variants
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
azurophil granuleextracellular exosomeN-acetylglucosamine-6-sulfatase activityheparan sulfate proteoglycan catabolic processmucopolysaccharidosis type 3Dmucopolysaccharidosis type 3mucopolysaccharidosispreeclampsia
✦AI Summary

GNS encodes N-acetylglucosamine-6-sulfatase, a lysosomal enzyme that hydrolyzes 6-sulfate groups from N-acetyl-D-glucosaminide units in heparan sulfate and keratan sulfate 1. This enzyme functions as a sulfuric ester hydrolase critical for glycosaminoglycan catabolism, participating in the sequential degradation of heparan sulfate within lysosomes 2. GNS mutations cause mucopolysaccharidosis type IIID (MPS IIID, Sanfilippo syndrome subtype D), a lysosomal storage disorder characterized by progressive heparan sulfate accumulation in lysosomes and extracellular compartments 1. This leads to secondary storage of glycosphingolipids, oxidative stress, and neuroinflammation, predominantly affecting the central nervous system with progressive cognitive decline and severe hyperactivity 1. A case report identified a novel homozygous GNS truncating mutation in a family presenting with the unusual combination of mucopolysaccharidosis and retinal dystrophy, expanding the clinical spectrum of GNS-associated disease 3. Additionally, GNS mutations are recognized among carbohydrate-linked lysosomal storage diseases presenting cardiac complications 4. Currently, no established cure exists; however, enzyme replacement therapy and gene therapy show promise in clinical trials for managing MPS III 2.

Sources cited
1
GNS codes for N-acetylglucosamine-6-sulfatase involved in heparan sulfate degradation; GNS mutations cause MPS IIID with heparan sulfate accumulation and neuroinflammation
PMID: 27100513
2
MPS III is a lysosomal storage disorder; GNS gene mutations differentiate MPS IIID; enzyme replacement therapy and gene therapy are in clinical trials
PMID: 25851924
3
Homozygous truncating GNS mutation identified in family with novel combination of mucopolysaccharidosis and retinal dystrophy
PMID: 26355662
4
GNS mutations listed among carbohydrate-linked lysosomal storage diseases presenting with cardiac complications
PMID: 37239976
⚠Limited data available β€” This gene has 4 indexed publications. Summary and analysis may be incomplete.
Disease Associationsβ“˜21
mucopolysaccharidosis type 3DOpen Targets
0.82Strong
mucopolysaccharidosis type 3Open Targets
0.69Moderate
mucopolysaccharidosisOpen Targets
0.37Weak
preeclampsiaOpen Targets
0.31Weak
hair colorOpen Targets
0.29Weak
ventricular fibrillationOpen Targets
0.28Weak
genetic disorderOpen Targets
0.19Weak
cornea transplantationOpen Targets
0.02Suggestive
muscular diseaseOpen Targets
0.02Suggestive
neoplasmOpen Targets
0.02Suggestive
SepsisOpen Targets
0.01Suggestive
glioblastomaOpen Targets
0.01Suggestive
ganglioneuromaOpen Targets
0.01Suggestive
chronic obstructive pulmonary diseaseOpen Targets
0.01Suggestive
glioblastoma multiformeOpen Targets
0.01Suggestive
gliomaOpen Targets
0.01Suggestive
septic shockOpen Targets
0.01Suggestive
cystic fibrosisOpen Targets
0.01Suggestive
dry eye syndromeOpen Targets
0.01Suggestive
ovarian cancerOpen Targets
0.01Suggestive
Mucopolysaccharidosis 3DUniProt
Pathogenic Variants83
NM_002076.4(GNS):c.1249C>T (p.Gln417Ter)Pathogenic
Mucopolysaccharidosis, MPS-III-D|Sanfilippo syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 417
NM_002076.4(GNS):c.1225C>T (p.Arg409Ter)Pathogenic
Mucopolysaccharidosis, MPS-III-D|Sanfilippo syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 409
NM_002076.4(GNS):c.802del (p.Trp268fs)Likely pathogenic
Mucopolysaccharidosis, MPS-III-D|Sanfilippo syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 268
NM_002076.4(GNS):c.1393C>T (p.Gln465Ter)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2025β†’ Residue 465
NM_002076.4(GNS):c.714G>A (p.Trp238Ter)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2025β†’ Residue 238
NM_002076.4(GNS):c.1231dup (p.Asp411fs)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2025β†’ Residue 411
NM_002076.4(GNS):c.1019A>G (p.Lys340Arg)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2025β†’ Residue 340
NM_002076.4(GNS):c.1308+2T>CLikely pathogenic
Mucopolysaccharidosis, MPS-III-D|Sanfilippo syndrome|not provided
β˜…β˜…β˜†β˜†2025
NM_002076.4(GNS):c.355del (p.Ser119fs)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2025β†’ Residue 119
NM_002076.4(GNS):c.1063C>T (p.Arg355Ter)Pathogenic
Mucopolysaccharidosis, MPS-III-D|not provided|Sanfilippo syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 355
NM_002076.4(GNS):c.625-2A>GLikely pathogenic
Mucopolysaccharidosis, MPS-III-D|Sanfilippo syndrome
β˜…β˜…β˜†β˜†2025
NM_002076.4(GNS):c.4del (p.Arg2fs)Pathogenic
Mucopolysaccharidosis, MPS-III-D|Sanfilippo syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 2
NM_002076.4(GNS):c.506G>A (p.Trp169Ter)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2024β†’ Residue 169
NM_002076.4(GNS):c.1414C>T (p.Gln472Ter)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2024β†’ Residue 472
NM_002076.4(GNS):c.252+1G>TLikely pathogenic
Mucopolysaccharidosis, MPS-III-D|not provided
β˜…β˜…β˜†β˜†2024
NM_002076.4(GNS):c.257_258del (p.Val86fs)Pathogenic
Mucopolysaccharidosis, MPS-III-D|Sanfilippo syndrome
β˜…β˜…β˜†β˜†2024β†’ Residue 86
NM_002076.4(GNS):c.876-2A>GPathogenic
Mucopolysaccharidosis, MPS-III-D|not provided
β˜…β˜…β˜†β˜†2024
NM_002076.4(GNS):c.1098+1G>APathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2023
NM_002076.4(GNS):c.1200+1G>TLikely pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜…β˜†β˜†2022
NM_002076.4(GNS):c.379del (p.Gln127fs)Pathogenic
Mucopolysaccharidosis, MPS-III-D
β˜…β˜†β˜†β˜†2025β†’ Residue 127
View on ClinVar β†—
Related Genes
GLB1Protein interaction96%HEXBProtein interaction94%HEXAProtein interaction94%SUMF1Protein interaction80%GUSBProtein interaction72%CTSZProtein interaction71%
Tissue Expression6 tissues
Lung
100%
Heart
96%
Ovary
83%
Brain
71%
Liver
35%
Bone Marrow
24%
Gene Interaction Network
Click a node to explore
GNSGLB1HEXBHEXASUMF1GUSBCTSZ
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt P15586
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
1.54LoF Tolerant
pLIβ“˜
0.01Tolerant
Observed/Expected LoF0.80 [0.43–1.54]
RankingsWhere GNS stands among ~20K protein-coding genes
  • #8,319of 20,598
    Most Researched54
  • #898of 5,498
    Most Pathogenic Variants83 Β· top quartile
  • #15,408of 17,882
    Most Constrained (LOEUF)1.54
Genes detectedGNS
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Expanding the clinical, allelic, and locus heterogeneity of retinal dystrophies.
PMID: 26355662
Genet Med Β· 2016
1.00
2
Metabolic Cardiomyopathies and Cardiac Defects in Inherited Disorders of Carbohydrate Metabolism: A Systematic Review.
PMID: 37239976
Int J Mol Sci Β· 2023
0.90
3
Paraneoplastic Glomerular Diseases.
PMID: 35817519
Adv Chronic Kidney Dis Β· 2022
0.80
4
[Post-infectious glomerulonephritis].
PMID: 12746805
G Ital Nefrol Β· 2003
0.70
5
Sanfilippo syndrome: Overall review.
PMID: 25851924
Pediatr Int Β· 2015
0.60