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GeneE
26 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.
SPTLC1
serine palmitoyltransferase long chain base subunit 1
Chromosome 9 Β· 9q22.31
NCBI Gene: 10558Ensembl: ENSG00000090054.17HGNC: HGNC:11277UniProt: O15269
166PubMed Papers
22Diseases
0Drugs
14Pathogenic Variants
RESEARCH IMPACT
Trending
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
sphingolipid biosynthetic processceramide biosynthetic processpositive regulation of lipophagyserine C-palmitoyltransferase activityneuropathy, hereditary sensory and autonomic, type 1Ahereditary sensory and autonomic neuropathy type 1Charcot-Marie-Tooth diseaseamyotrophic lateral sclerosis 27, juvenile
✦AI Summary

SPTLC1 encodes serine palmitoyltransferase long chain base subunit 1, a critical component of the serine palmitoyltransferase (SPT) complex that catalyzes the initial and rate-limiting step of sphingolipid biosynthesis by condensing L-serine with activated acyl-CoA (primarily palmitoyl-CoA) to form long-chain bases 1. SPTLC1 forms the catalytic core when paired with SPTLC2 or SPTLC3, with complex composition determining substrate specificity; the SPTLC1-SPTLC2-SPTSSA complex preferentially uses C16-CoA substrate, while SPTLC1-SPTLC2-SPTSSB shows strong C18-CoA preference 1. Under low serine availability, SPT can metabolize L-alanine instead, producing pathogenic 1-deoxysphingolipids 2. De novo SPTLC1 variants cause juvenile amyotrophic lateral sclerosis (ALS) with failure to thrive, presenting with elevated sphinganine and ceramide levels 34. C-terminal SPTLC1 variants cause hereditary sensory and autonomic neuropathy type 1A (HSAN1) through 1-deoxysphingolipid accumulation 2. Clinical phenotype depends on variant location and serine availabilityβ€”ALS-associated variants impair ORMDL protein binding, increasing canonical sphingolipid synthesis, while substrate-level modulation can shift phenotypes 2. Reduced SPTLC1 expression variants associate with improved fitness in aging, suggesting therapeutic potential for age-related sarcopenia 5.

Sources cited
1
SPT catalyzes de novo sphingolipid biosynthesis by condensing L-serine and activated acyl-CoA; substrate specificity determined by complex composition
PMID: 32788725
2
De novo SPTLC1 variants (p.Ala20Ser, p.Ser331Tyr, p.Leu39del) cause juvenile ALS with failure to thrive; variants disrupt sphingolipid synthesis pathway
PMID: 34459874
3
SPTLC1 variants associated with juvenile ALS cause elevated sphinganine and ceramide levels; pathogenic c.58G>A variant identified in juvenile ALS patient
PMID: 39666121
4
ALS-associated SPTLC1 variants impair ORMDL binding causing increased sphingolipid synthesis; C-terminal variants cause HSAN1 via 1-deoxysphingolipid synthesis; serine availability modulates clinical phenotype
PMID: 35900868
5
SPTLC1 expression-reducing variants associate with improved fitness in older individuals; sphingolipid synthesis inhibition prevents age-related muscle decline
PMID: 37118545
Disease Associationsβ“˜22
neuropathy, hereditary sensory and autonomic, type 1AOpen Targets
0.67Moderate
hereditary sensory and autonomic neuropathy type 1Open Targets
0.67Moderate
Charcot-Marie-Tooth diseaseOpen Targets
0.58Moderate
amyotrophic lateral sclerosis 27, juvenileOpen Targets
0.51Moderate
Familial dysautonomiaOpen Targets
0.46Moderate
hereditary sensory and autonomic neuropathy type 2Open Targets
0.46Moderate
Riley-Day syndromeOpen Targets
0.46Moderate
genetic disorderOpen Targets
0.45Moderate
juvenile amyotrophic lateral sclerosisOpen Targets
0.38Weak
hereditary sensory neuropathy-deafness-dementia syndromeOpen Targets
0.37Weak
neurodegenerative diseaseOpen Targets
0.35Weak
Ritscher-Schinzel syndrome 4Open Targets
0.34Weak
Sensorimotor neuropathyOpen Targets
0.33Weak
amyotrophic lateral sclerosisOpen Targets
0.32Weak
Autosomal dominant familial hematuria - retinal arteriolar tortuosity - contracturesOpen Targets
0.27Weak
EMG abnormalityOpen Targets
0.27Weak
FallsOpen Targets
0.27Weak
Muscle spasmOpen Targets
0.27Weak
Proximal lower limb amyotrophyOpen Targets
0.27Weak
Proximal muscle weaknessOpen Targets
0.27Weak
Amyotrophic lateral sclerosis 27, juvenileUniProt
Neuropathy, hereditary sensory and autonomic, 1AUniProt
Pathogenic Variants14
NM_006415.4(SPTLC1):c.431T>A (p.Val144Asp)Pathogenic
Hereditary sensory and autonomic neuropathy type 1|not provided|Charcot-Marie-Tooth disease|Neuropathy, hereditary sensory and autonomic, type 1A|Inborn genetic diseases|SPTLC1-related disorder|Amyotrophic lateral sclerosis 27, juvenile
β˜…β˜…β˜†β˜†2026β†’ Residue 144
NM_006415.4(SPTLC1):c.399T>G (p.Cys133Trp)Pathogenic
Hereditary sensory and autonomic neuropathy type 1|Charcot-Marie-Tooth disease|Sensorimotor neuropathy|Neuropathy, hereditary sensory and autonomic, type 1A|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 133
NM_006415.4(SPTLC1):c.398G>A (p.Cys133Tyr)Pathogenic
Hereditary sensory and autonomic neuropathy type 1|Neuropathy, hereditary sensory and autonomic, type 1A|Charcot-Marie-Tooth disease|Inborn genetic diseases|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 133
NM_006415.4(SPTLC1):c.58G>T (p.Ala20Ser)Pathogenic
not provided|Amyotrophic lateral sclerosis 27, juvenile|Neuropathy, hereditary sensory and autonomic, type 1A|Ritscher-Schinzel syndrome 4
β˜…β˜…β˜†β˜†2025β†’ Residue 20
NM_006415.4(SPTLC1):c.112CTT[1] (p.Leu39del)Pathogenic
Neuropathy, hereditary sensory and autonomic, type 1A|not provided|Hereditary sensory and autonomic neuropathy type 1|Proximal lower limb amyotrophy;Muscle spasm;EMG abnormality;Falls;Proximal muscle weakness|Amyotrophic lateral sclerosis 27, juvenile
β˜…β˜…β˜†β˜†2023β†’ Residue 39
NM_006415.4(SPTLC1):c.992C>A (p.Ser331Tyr)Pathogenic
not provided|Charcot-Marie-Tooth disease|Hereditary sensory and autonomic neuropathy type 1|Neuropathy, hereditary sensory and autonomic, type IA, severe|Amyotrophic lateral sclerosis 27, juvenile
β˜…β˜…β˜†β˜†2022β†’ Residue 331
NM_006415.4(SPTLC1):c.68A>T (p.Tyr23Phe)Likely pathogenic
not provided|Neuropathy, hereditary sensory and autonomic, type 1A|Amyotrophic lateral sclerosis 27, juvenile
β˜…β˜…β˜†β˜†2020β†’ Residue 23
NM_006415.4(SPTLC1):c.1015G>T (p.Ala339Ser)Pathogenic
Hereditary sensory and autonomic neuropathy type 1
β˜…β˜†β˜†β˜†2025β†’ Residue 339
NM_006415.4(SPTLC1):c.992C>T (p.Ser331Phe)Pathogenic
Neuropathy, hereditary sensory and autonomic, type IA, severe|Neuropathy, hereditary sensory and autonomic, type 1A
β˜…β˜†β˜†β˜†2023β†’ Residue 331
NM_006415.4(SPTLC1):c.398G>T (p.Cys133Phe)Likely pathogenic
Hereditary sensory and autonomic neuropathy type 1
β˜…β˜†β˜†β˜†2022β†’ Residue 133
NM_006415.4(SPTLC1):c.986G>A (p.Arg329Gln)Likely pathogenic
Neuropathy, hereditary sensory and autonomic, type 1A
β˜…β˜†β˜†β˜†2021β†’ Residue 329
NM_006415.4(SPTLC1):c.1072G>C (p.Glu358Gln)Likely pathogenic
Hereditary sensory and autonomic neuropathy type 1
β˜…β˜†β˜†β˜†2014β†’ Residue 358
NM_006415.4(SPTLC1):c.118_123del (p.Phe40_Ser41del)Likely pathogenic
Amyotrophic lateral sclerosis|Amyotrophic lateral sclerosis 27, juvenile
β˜…β˜†β˜†β˜†β†’ Residue 40
NM_006415.4(SPTLC1):c.113T>G (p.Leu38Arg)Pathogenic
Amyotrophic lateral sclerosis 27, juvenile
β˜†β˜†β˜†β˜†2023β†’ Residue 38
View on ClinVar β†—
Related Genes
AGXTProtein interaction100%CBSProtein interaction100%GDF1Protein interaction100%ORM1Protein interaction100%PSPHProtein interaction100%SHMT1Protein interaction100%
Tissue Expression6 tissues
Bone Marrow
100%
Brain
96%
Heart
70%
Ovary
66%
Lung
50%
Liver
29%
Gene Interaction Network
Click a node to explore
SPTLC1AGXTCBSGDF1ORM1PSPHSHMT1
PROTEIN STRUCTURE
Preparing viewer…
PDB7K0K Β· 2.60 Γ… Β· EM
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
1.07LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.81 [0.62–1.07]
RankingsWhere SPTLC1 stands among ~20K protein-coding genes
  • #2,700of 20,598
    Most Researched166 Β· top quartile
  • #2,503of 5,498
    Most Pathogenic Variants14
  • #10,770of 17,882
    Most Constrained (LOEUF)1.07
Genes detectedSPTLC1
Sources retrieved26 papers
Response timeβ€”
πŸ“„ Sources
26β–Ό
1
A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort.
PMID: 34602496
J Neuromuscul Dis Β· 2022
1.00
2
Sphingolipids accumulate in aged muscle, and their reduction counteracts sarcopenia.
PMID: 37118545
Nat Aging Β· 2022
0.90
3
Serine restriction alters sphingolipid diversity to constrain tumour growth.
PMID: 32788725
Nature Β· 2020
0.80
4
Association of Variants in the SPTLC1 Gene With Juvenile Amyotrophic Lateral Sclerosis.
PMID: 34459874
JAMA Neurol Β· 2021
0.70
5
Mutation screening of SPTLC1 and SPTLC2 in amyotrophic lateral sclerosis.
PMID: 36966328
Hum Genomics Β· 2023
0.68