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10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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CARS1
cysteinyl-tRNA synthetase 1
Chromosome 11 Β· 11p15.4
NCBI Gene: 833Ensembl: ENSG00000110619.18HGNC: HGNC:1493UniProt: B4DKY1
73PubMed Papers
21Diseases
0Drugs
7Pathogenic Variants
FUNCTIONAL ROLE
Oncogene
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
tRNA bindingidentical protein bindingcysteinyl-tRNA aminoacylationcytoplasmmicrocephaly, developmental delay, and brittle hair syndromecancertrichothiodystrophyskin squamous cell carcinoma
✦AI Summary

CARS1 (cysteinyl-tRNA synthetase 1) catalyzes the ATP-dependent aminoacylation of cysteine to tRNA(Cys), a critical step in protein synthesis 1. The enzyme functions as a cytoplasmic aminoacyl-tRNA synthetase with established roles in translation initiation and elongation. Loss-of-function CARS1 variants cause a multisystem neurodevelopmental disorder characterized by microcephaly, developmental delay, brittle hair and nails, and variable additional manifestations 12. Disease severity correlates with variant functional impact; compound heterozygous mutations can produce hypomorphic or complete loss-of-function effects 1. The phenotypic spectrum has expanded to include hepatopathy, hypothyroidism, muscular hypotonia, ataxia, and congenital hyperinsulinism, indicating broader metabolic involvement 2. Beyond monogenic disease, CARS1 has emerging roles in complex disorders. A recurrent de novo missense variant (p.E712V) associates with sporadic moyamoya disease, exhibiting early-onset cerebrovascular stenosis in affected carriers 3. Additionally, CARS1 contains an endogenous TLR2/6 ligand domain with immunomodulatory properties capable of activating dendritic cells and enhancing anti-tumor immunity 4. CARS1 is also implicated as an autoantigen in antisynthetase syndrome and serves as a prognostic marker in mesothelioma 56.

Sources cited
1
Loss-of-function CARS1 variants cause microcephaly, developmental delay, brittle hair phenotype, and hepatopathy; yeast complementation assays demonstrated hypomorphic and complete loss-of-function effects
PMID: 39963003
2
CARS1 variants expand phenotype to include congenital hyperinsulinism alongside neurodevelopmental and multisystem features
PMID: 41121266
3
Recurrent CARS1 p.E712V variant associates with sporadic moyamoya disease with early-onset stenosis; functional studies showed enhanced angiogenesis in endothelial cells
PMID: 41605952
4
CARS1 contains endogenous TLR2/6 ligand domain (UNE-C1) capable of activating dendritic cells and stimulating immune responses with immunoadjuvant properties
PMID: 32461342
5
Cytoplasmic CARS1 identified as autoantigen in antisynthetase syndrome with anti-Ly autoantibody specificity
PMID: 36572507
6
Elevated CARS1 expression associated with unfavorable prognosis in mesothelioma and included in ferroptosis-related prognostic signature
PMID: 38670397
Disease Associationsβ“˜21
microcephaly, developmental delay, and brittle hair syndromeOpen Targets
0.67Moderate
cancerOpen Targets
0.47Moderate
trichothiodystrophyOpen Targets
0.37Weak
breast ductal adenocarcinomaOpen Targets
0.37Weak
carcinoma of liver and intrahepatic biliary tractOpen Targets
0.37Weak
colorectal adenocarcinomaOpen Targets
0.37Weak
cutaneous melanomaOpen Targets
0.37Weak
Endometrial Endometrioid AdenocarcinomaOpen Targets
0.37Weak
hemangioblastomaOpen Targets
0.37Weak
kidney neoplasmOpen Targets
0.37Weak
lung carcinomaOpen Targets
0.37Weak
lymphoid neoplasmOpen Targets
0.37Weak
skin squamous cell carcinomaOpen Targets
0.37Weak
esophageal squamous cell carcinomaOpen Targets
0.30Weak
Abruptio PlacentaeOpen Targets
0.29Weak
hepatocellular carcinomaOpen Targets
0.29Weak
clear cell renal carcinomaOpen Targets
0.28Weak
inflammatory myofibroblastic tumorOpen Targets
0.28Weak
acute myeloid leukemiaOpen Targets
0.28Weak
melanomaOpen Targets
0.28Weak
Microcephaly, developmental delay, and brittle hair syndromeUniProt
Pathogenic Variants7
NM_001014437.3(CARS1):c.234_237delinsCAAAGGAGCTTT (p.Ser79fs)Pathogenic
Microcephaly, developmental delay, and brittle hair syndrome
β˜…β˜†β˜†β˜†2023β†’ Residue 79
NM_001014437.3(CARS1):c.1271G>A (p.Arg424His)Likely pathogenic
Microcephaly, developmental delay, and brittle hair syndrome
β˜…β˜†β˜†β˜†2021β†’ Residue 424
NM_001014437.3(CARS1):c.451G>A (p.Ala151Thr)Likely pathogenic
Microcephaly, developmental delay, and brittle hair syndrome
β˜…β˜†β˜†β˜†β†’ Residue 151
NM_001014437.3(CARS1):c.1325C>T (p.Ser442Leu)Pathogenic
Microcephaly, developmental delay, and brittle hair syndrome
β˜†β˜†β˜†β˜†2021β†’ Residue 442
NM_001014437.3(CARS1):c.1387C>T (p.Gln463Ter)Pathogenic
Microcephaly, developmental delay, and brittle hair syndrome
β˜†β˜†β˜†β˜†2021β†’ Residue 463
NM_001014437.3(CARS1):c.2310dup (p.Ser771fs)Pathogenic
Microcephaly, developmental delay, and brittle hair syndrome
β˜†β˜†β˜†β˜†2021β†’ Residue 771
NM_001014437.3(CARS1):c.1448T>A (p.Leu483Gln)Pathogenic
Microcephaly, developmental delay, and brittle hair syndrome
β˜†β˜†β˜†β˜†2021β†’ Residue 483
View on ClinVar β†—
Related Genes
LARS1Protein interaction100%QARS1Protein interaction100%KARS1Protein interaction100%EPRS1Protein interaction100%IARS1Protein interaction100%EEF1A1Protein interaction99%
Tissue Expression6 tissues
Lung
100%
Brain
92%
Liver
89%
Bone Marrow
43%
Heart
40%
Ovary
39%
Gene Interaction Network
Click a node to explore
CARS1LARS1QARS1KARS1EPRS1IARS1EEF1A1
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt P49589
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.57Moderately Constrained
pLIβ“˜
0.01Tolerant
Observed/Expected LoF0.44 [0.34–0.57]
RankingsWhere CARS1 stands among ~20K protein-coding genes
  • #6,450of 20,598
    Most Researched73
  • #3,197of 5,498
    Most Pathogenic Variants7
  • #3,763of 17,882
    Most Constrained (LOEUF)0.57 Β· top quartile
Genes detectedCARS1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Mass spectrometry-based identification of new anti-Ly and known antisynthetase autoantibodies.
PMID: 36572507
Ann Rheum Dis Β· 2023
1.00
2
Loss-of-Function CARS1 Variants in a Patient With Microcephaly, Developmental Delay, and a Brittle Hair Phenotype.
PMID: 39963003
Mol Genet Genomic Med Β· 2025
0.90
3
Evaluation of Exosome-derived Small RNAs as Potential Biomarkers for Pancreatic Ductal Adenocarcinoma Using Next-generation Sequencing.
PMID: 40958715
Ann Lab Med Β· 2025
0.80
4
Identification and validation of CARS1 p.E712V and NF1 p.Q2002X in sporadic Moyamoya disease across 30 trio pedigrees.
PMID: 41605952
NPJ Genom Med Β· 2026
0.70
5
Endogenous TLR2 ligand embedded in the catalytic region of human cysteinyl-tRNA synthetase 1.
PMID: 32461342
J Immunother Cancer Β· 2020
0.60