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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.
DEGS1
delta 4-desaturase, sphingolipid 1
Chromosome 1 Β· 1q42.11
NCBI Gene: 8560Ensembl: ENSG00000143753.15HGNC: HGNC:13709UniProt: O15121
56PubMed Papers
21Diseases
0Drugs
20Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
retinol isomerase activitymyelin maintenanceceramide biosynthetic processcytoplasmic side of endoplasmic reticulum membraneleukodystrophy, hypomyelinating, 18leukodystrophygenetic disorderdevelopmental disability
✦AI Summary

DEGS1 encodes delta 4-desaturase, sphingolipid 1, a key enzyme in sphingolipid biosynthesis that catalyzes the conversion of dihydroceramide to ceramide by introducing a double bond at the C4-C5 position 1. The enzyme is localized to mitochondria-associated endoplasmic reticulum membranes (MAM) where it plays essential roles in maintaining MAM integrity and function 1. DEGS1 deficiency disrupts multiple MAM functions including mitochondrial dynamics, cholesterol metabolism, phospholipid metabolism, and lipid droplet biogenesis, while also impairing mitochondrial respiration 1. Biallelic pathogenic variants in DEGS1 cause hypomyelinating leukodystrophy-18 (HLD18), a rare neurodevelopmental disorder characterized by severe developmental delay, failure to thrive, dystonia, seizures, and brain hypomyelination 2. Functional assessment shows significantly elevated dihydroceramide/ceramide ratios in patient samples, confirming enzyme dysfunction 2. Beyond its role in disease, DEGS1 has broader physiological significance, with genetic variants associated with fitness outcomes in aging populations 3 and the enzyme playing regulatory roles in hematopoietic stem cell self-renewal through sphingolipid modulation 4. The enzyme is also responsive to inflammatory cytokines in keratinocytes, suggesting involvement in skin barrier function 5.

Sources cited
1
DEGS1 is a MAM-resident enzyme that converts dihydroceramide to ceramide and is essential for MAM integrity and mitochondrial function
PMID: 36951944
2
Biallelic DEGS1 variants cause hypomyelinating leukodystrophy-18 with elevated dihydroceramide/ceramide ratios
PMID: 37195341
3
DEGS1 genetic variants are associated with fitness outcomes in aging populations
PMID: 37118545
4
DEGS1 regulates hematopoietic stem cell self-renewal through sphingolipid modulation
PMID: 31631013
5
DEGS1 expression is regulated by inflammatory cytokines in keratinocytes
PMID: 40046180
Disease Associationsβ“˜21
leukodystrophy, hypomyelinating, 18Open Targets
0.76Strong
leukodystrophyOpen Targets
0.72Strong
genetic disorderOpen Targets
0.38Weak
developmental disabilityOpen Targets
0.37Weak
SeizureOpen Targets
0.37Weak
neurodegenerative diseaseOpen Targets
0.28Weak
Phenotypic abnormalityOpen Targets
0.08Suggestive
alcohol drinkingOpen Targets
0.08Suggestive
poisoningOpen Targets
0.06Suggestive
oral squamous cell carcinomaOpen Targets
0.06Suggestive
lymphatic malformation 11Open Targets
0.05Suggestive
neonatal intrahepatic cholestasis due to citrin deficiencyOpen Targets
0.04Suggestive
hereditary angioedemaOpen Targets
0.04Suggestive
neoplasmOpen Targets
0.04Suggestive
hereditary angioedema with normal C1InhOpen Targets
0.04Suggestive
adverse effectOpen Targets
0.04Suggestive
gastrointestinal diseaseOpen Targets
0.04Suggestive
response to stimulusOpen Targets
0.04Suggestive
LipedemaOpen Targets
0.04Suggestive
lymphatic malformation 8Open Targets
0.04Suggestive
Leukodystrophy, hypomyelinating, 18UniProt
Pathogenic Variants20
NM_003676.4(DEGS1):c.517C>T (p.Arg173Ter)Pathogenic
Leukodystrophy, hypomyelinating, 18|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 173
NM_003676.4(DEGS1):c.775C>T (p.His259Tyr)Likely pathogenic
Leukodystrophy, hypomyelinating, 18|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 259
NM_003676.4(DEGS1):c.752dup (p.Leu251fs)Likely pathogenic
Inborn genetic diseases|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 251
NM_003676.4(DEGS1):c.337A>G (p.Asn113Asp)Pathogenic
Leukodystrophy, hypomyelinating, 18|DEGS1-related Hypomyelinating Leukodystrophy
β˜…β˜…β˜†β˜†2025β†’ Residue 113
NM_003676.4(DEGS1):c.764A>G (p.Asn255Ser)Pathogenic
Leukodystrophy, hypomyelinating, 18|Leukodystrophy|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 255
NM_003676.4(DEGS1):c.852_855del (p.Tyr283_Tyr284insTer)Pathogenic
Leukodystrophy, hypomyelinating, 18
β˜…β˜…β˜†β˜†2023β†’ Residue 283
NM_003676.4(DEGS1):c.320G>A (p.Trp107Ter)Pathogenic
Leukodystrophy, hypomyelinating, 18
β˜…β˜…β˜†β˜†2022β†’ Residue 107
NM_003676.4(DEGS1):c.397C>T (p.Arg133Trp)Likely pathogenic
Leukodystrophy, hypomyelinating, 18|Inborn genetic diseases
β˜…β˜…β˜†β˜†2019β†’ Residue 133
NM_003676.4(DEGS1):c.201A>T (p.Lys67Asn)Likely pathogenic
DEGS1-related Hypomyelinating Leukodystrophy
β˜…β˜†β˜†β˜†2025β†’ Residue 67
NM_003676.4(DEGS1):c.825+4_825+5delinsTTPathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_003676.4(DEGS1):c.252dup (p.Thr85fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 85
Single allelePathogenic
Leukodystrophy, hypomyelinating, 18
β˜…β˜†β˜†β˜†2024
NM_003676.4(DEGS1):c.22G>T (p.Glu8Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2023β†’ Residue 8
NM_003676.4(DEGS1):c.49C>T (p.Gln17Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2022β†’ Residue 17
NM_003676.4(DEGS1):c.82+1G>CLikely pathogenic
not provided
β˜…β˜†β˜†β˜†2022
NM_003676.4(DEGS1):c.839C>T (p.Ala280Val)Likely pathogenic
Leukodystrophy, hypomyelinating, 18
β˜…β˜†β˜†β˜†2019β†’ Residue 280
NM_003676.4(DEGS1):c.2T>C (p.Met1Thr)Likely pathogenic
Leukodystrophy, hypomyelinating, 18
β˜…β˜†β˜†β˜†β†’ Residue 1
NM_003676.4(DEGS1):c.826-1G>ALikely pathogenic
Leukodystrophy, hypomyelinating, 18
β˜…β˜†β˜†β˜†
NM_003676.4(DEGS1):c.604del (p.Tyr202fs)Pathogenic
Leukodystrophy, hypomyelinating, 18
β˜†β˜†β˜†β˜†2019β†’ Residue 202
NM_003676.4(DEGS1):c.341_342del (p.Leu114fs)Pathogenic
Leukodystrophy, hypomyelinating, 18
β˜†β˜†β˜†β˜†2019β†’ Residue 114
View on ClinVar β†—
Related Genes
ASAH2Protein interaction98%ACER2Protein interaction97%ACER1Protein interaction97%CERS6Protein interaction97%CERS2Protein interaction97%CERS4Protein interaction97%
Tissue Expression6 tissues
Bone Marrow
100%
Brain
79%
Heart
72%
Lung
70%
Ovary
38%
Liver
37%
Gene Interaction Network
Click a node to explore
DEGS1ASAH2ACER2ACER1CERS6CERS2CERS4
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt O15121
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
1.05LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.71 [0.49–1.05]
RankingsWhere DEGS1 stands among ~20K protein-coding genes
  • #8,058of 20,598
    Most Researched56
  • #2,199of 5,498
    Most Pathogenic Variants20
  • #10,521of 17,882
    Most Constrained (LOEUF)1.05
Genes detectedDEGS1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
SCORE2-Diabetes: 10-year cardiovascular risk estimation in type 2 diabetes in Europe.
PMID: 37247330
Eur Heart J Β· 2023
1.00
2
Sphingolipids accumulate in aged muscle, and their reduction counteracts sarcopenia.
PMID: 37118545
Nat Aging Β· 2022
0.90
3
Burden of disease in myasthenia gravis: taking the patient's perspective.
PMID: 34800167
J Neurol Β· 2022
0.80
4
Sphingolipid desaturase DEGS1 is essential for mitochondria-associated membrane integrity.
PMID: 36951944
J Clin Invest Β· 2023
0.70
5
DEGS1 -related leukodystrophy: a clinical report and review of literature.
PMID: 37195341
Clin Dysmorphol Β· 2023
0.60