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GeneE
9 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.
RARS1
arginyl-tRNA synthetase 1
Chromosome 5 Β· 5q34
NCBI Gene: 5917Ensembl: ENSG00000113643.10HGNC: HGNC:9870UniProt: P54136
241PubMed Papers
21Diseases
0Drugs
35Pathogenic Variants
FUNCTIONAL ROLE
Hub Gene
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
cytoplasmcytosolmembraneextracellular exosomehypomyelinating leukodystrophy 9Hypomyelinating leukodystrophy with or without oligondontia and/or hypogonadismgenetic disorderleukodystrophy
✦AI Summary

RARS1 encodes arginyl-tRNA synthetase 1 (ArgRS), a cytoplasmic enzyme that catalyzes the attachment of arginine to cognate tRNAs during protein synthesis 1. RARS1 functions as a core component of the multi-tRNA synthetase complex (MSC), assembling with AIMPs through leucine zipper motifs to form a structured macromolecular complex 2. Beyond translation, RARS1 modulates secretion of AIMP1 and may regulate generation of the inflammatory cytokine EMAP2 from AIMP1 1. Biallelic RARS1 variants cause hypomyelinating leukodystrophy-9 (HLD-9) and developmental and epileptic encephalopathy (DEE) 3. Disease severity correlates with variant type: truncating variants and those affecting the ArgRS active center cause severe early-onset epilepsy with brain atrophy, while the recurrent c.5A>G mutation produces milder phenotypes 4. Pathogenic variants reduce ArgRS protein stability or activity, impairing translation of ArgRS isoforms 43. Notably, the c.5A>G mutation suppresses mRNA translation through an upstream open reading frame mechanism rather than reducing protein stability 5. Common clinical features include intellectual disability, developmental delay, hypomyelination, nystagmus, and spasticity, with disease onset typically between 0-10 months 6. Beyond white matter effects, RARS1 mutations influence cortical development 3. Genetic screening enables diagnosis in approximately half of patients presenting with hypomyelinating leukoencephalopathy 7.

Sources cited
1
RARS1 catalyzes aminoacyl-tRNA synthesis and modulates AIMP1 secretion/EMAP2 generation
PMID: 25288775
2
RARS1 assembles into the multi-tRNA synthetase complex through leucine zipper motifs with AIMPs
PMID: 39542129
3
Biallelic RARS1 mutations cause DEE and HLD-9; pathogenic variants reduce ArgRS protein stability and expression
PMID: 37186453
4
RARS1 variants produce phenotype spectrum from severe epileptic encephalopathy to mild disease; genotype-phenotype correlations exist
PMID: 31814314
5
RARS1 pathogenic variants cause HLD-9 with typical onset 0-10 months; c.5A>G is most common variant
PMID: 38618971
6
c.5A>G mutation impairs RARS1 mRNA translation through upstream open reading frame, not protein stability
PMID: 33515434
7
Genetic screening identifies RARS1 variants in ~44% of adult hypomyelinating leukoencephalopathy cases
PMID: 33190326
Disease Associationsβ“˜21
hypomyelinating leukodystrophy 9Open Targets
0.74Strong
Hypomyelinating leukodystrophy with or without oligondontia and/or hypogonadismOpen Targets
0.55Moderate
genetic disorderOpen Targets
0.47Moderate
leukodystrophyOpen Targets
0.27Weak
neoplasmOpen Targets
0.08Suggestive
Abnormality of the skeletal systemOpen Targets
0.08Suggestive
hyperaldosteronismOpen Targets
0.05Suggestive
gastric adenocarcinomaOpen Targets
0.05Suggestive
joint diseaseOpen Targets
0.05Suggestive
diabetes mellitusOpen Targets
0.03Suggestive
myelodysplastic syndromeOpen Targets
0.03Suggestive
multiple sclerosisOpen Targets
0.02Suggestive
multinodular goiterOpen Targets
0.02Suggestive
acute tonsillitisOpen Targets
0.02Suggestive
Varicose veinsOpen Targets
0.02Suggestive
adverse effectOpen Targets
0.02Suggestive
response to stimulusOpen Targets
0.02Suggestive
cervical carcinomaOpen Targets
0.02Suggestive
cancerOpen Targets
0.02Suggestive
ovarian neoplasmOpen Targets
0.02Suggestive
Leukodystrophy, hypomyelinating, 9UniProt
Pathogenic Variants35
NM_002887.4(RARS1):c.175C>T (p.Arg59Ter)Pathogenic
not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 59
NM_002887.4(RARS1):c.5A>G (p.Asp2Gly)Pathogenic
Hypomyelinating leukodystrophy 9|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 2
NM_002887.4(RARS1):c.1535G>A (p.Arg512Gln)Pathogenic
Hypomyelinating leukodystrophy 9|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 512
NM_002887.4(RARS1):c.1716G>A (p.Trp572Ter)Likely pathogenic
Hypomyelinating leukodystrophy 9
β˜…β˜…β˜†β˜†2024β†’ Residue 572
NM_002887.4(RARS1):c.1316C>A (p.Ala439Asp)Likely pathogenic
Hypomyelinating leukodystrophy 9|Leukodystrophy
β˜…β˜…β˜†β˜†2024β†’ Residue 439
NM_002887.4(RARS1):c.1452+1G>APathogenic
not provided|Hypomyelinating leukodystrophy 9
β˜…β˜…β˜†β˜†2024
NM_002887.4(RARS1):c.1443_1446del (p.Arg482fs)Pathogenic
not provided|Inborn genetic diseases
β˜…β˜…β˜†β˜†2024β†’ Residue 482
NM_002887.4(RARS1):c.2T>C (p.Met1Thr)Pathogenic
not provided|Hypomyelinating leukodystrophy 9|Inborn genetic diseases
β˜…β˜…β˜†β˜†2024β†’ Residue 1
NM_002887.4(RARS1):c.1A>G (p.Met1Val)Pathogenic
Hypomyelinating leukodystrophy 9|not provided
β˜…β˜…β˜†β˜†2022β†’ Residue 1
NM_002887.4(RARS1):c.160C>T (p.Arg54Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 54
NM_002887.4(RARS1):c.369+1G>ALikely pathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_002887.4(RARS1):c.1581_1582del (p.Gly528fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 528
NM_002887.4(RARS1):c.748_754del (p.Ala250fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 250
NM_002887.4(RARS1):c.579G>T (p.Lys193Asn)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 193
NM_002887.4(RARS1):c.6_7delinsGA (p.Asp2_Val3delinsGluIle)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 2
NM_002887.4(RARS1):c.180+2T>CLikely pathogenic
not provided
β˜…β˜†β˜†β˜†2024
NM_002887.4(RARS1):c.999del (p.Glu335fs)Pathogenic
Hypomyelinating leukodystrophy 9
β˜…β˜†β˜†β˜†2024β†’ Residue 335
NM_002887.4(RARS1):c.49G>T (p.Glu17Ter)Pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 17
NM_002887.4(RARS1):c.1579dup (p.Arg527fs)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 527
NM_002887.4(RARS1):c.1283G>A (p.Trp428Ter)Likely pathogenic
not provided
β˜…β˜†β˜†β˜†2023β†’ Residue 428
View on ClinVar β†—
Related Genes
AIMP1Protein interaction100%EEF1E1Protein interaction100%AARS1Protein interaction100%IARS1Protein interaction100%KARS1Protein interaction100%MARS1Protein interaction100%
Tissue Expression6 tissues
Heart
100%
Liver
90%
Lung
83%
Bone Marrow
66%
Ovary
64%
Brain
60%
Gene Interaction Network
Click a node to explore
RARS1AIMP1EEF1E1AARS1IARS1KARS1MARS1
PROTEIN STRUCTURE
Preparing viewer…
PDB4ZAJ Β· 2.22 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
1.08LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.86 [0.69–1.08]
RankingsWhere RARS1 stands among ~20K protein-coding genes
  • #1,630of 20,598
    Most Researched241 Β· top 10%
  • #1,666of 5,498
    Most Pathogenic Variants35
  • #10,941of 17,882
    Most Constrained (LOEUF)1.08
Genes detectedRARS1
Sources retrieved9 papers
Response timeβ€”
πŸ“„ Sources
9β–Ό
1
RARS1-related developmental and epileptic encephalopathy.
PMID: 37186453
Epilepsia Open Β· 2023
1.00
2
Assembly of the Human Multi-tRNA Synthetase Complex Through Leucine Zipper Motifs.
PMID: 39542129
J Mol Biol Β· 2024
0.89
3
RARS1-related hypomyelinating leukodystrophy: Expanding the spectrum.
PMID: 31814314
Ann Clin Transl Neurol Β· 2020
0.78
4
Overview of genetic variants in a cohort of Iranian patients with leukodystrophy.
PMID: 40594583
Sci Rep Β· 2025
0.67
5
RARS1-related hypomyelinating leukodystrophy-9 (HLD-9) in two distinct Iranian families: Case report and literature review.
PMID: 38618971
Mol Genet Genomic Med Β· 2024
0.56