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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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RARS2
arginyl-tRNA synthetase 2, mitochondrial
Chromosome 6 Β· 6q15
NCBI Gene: 57038Ensembl: ENSG00000146282.19HGNC: HGNC:21406UniProt: A0A8I5KPZ0
60PubMed Papers
21Diseases
0Drugs
242Pathogenic Variants
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
RNA bindingmitochondrionmitochondrial membranearginine-tRNA ligase activitypontocerebellar hypoplasia type 6pontocerebellar hypoplasiaNon-syndromic pontocerebellar hypoplasiagenetic disorder
✦AI Summary

RARS2 encodes mitochondrial arginyl-tRNA synthetase 2, which catalyzes the attachment of arginine to tRNA(Arg) in a two-step reaction essential for mitochondrial protein synthesis 1. The enzyme first activates arginine with ATP to form Arg-AMP, then transfers it to the acceptor end of tRNA(Arg) 1. RARS2 is a nuclear-encoded protein imported into mitochondria after cytosolic translation 2. RARs2 pathogenic variants cause pontocerebellar hypoplasia type 6 (PCH6), an autosomal recessive mitochondrial encephalopathy 34. The phenotypic spectrum has expanded to include early-onset developmental and epileptic encephalopathy (DEE), infantile myoclonic encephalopathy, and Lennox-Gastaut syndrome 51. Notably, 85% of reported RARS2 variants disrupt splicing or gene expression, leading to protein truncation 5. Clinical features include progressive microcephaly, hypotonia, seizures (often pharmacoresistant), failure to thrive, and developmental delay 1. Some patients develop hypoglycemia and lactic acidosis 6. Pathogenic mechanisms involve defective mitochondrial translation and dysregulated cellular physiology, including elevated oxidative stress and apoptosis, rather than primary energy metabolism defects 4. Yeast models confirm pathogenicity of missense variants and suggest arginine supplementation as potential therapeutic intervention 2. Ketogenic diet may reduce seizure frequency 1.

Sources cited
1
RARS2 catalyzes arginine transfer to tRNA(Arg) for mitochondrial protein synthesis; classical and expanded clinical phenotypes including DEE and LGS
PMID: 38438854
2
RARS2 encodes mitochondrial arginyl-tRNA synthetase; variant functional assessment in yeast; arginine supplementation as potential therapy
PMID: 39230874
3
RARS2 variants in Kozak sequence cause PCH6; impact on both transcription and protein translation
PMID: 37344844
4
PCH6 caused by RARS2 mutations; mitochondrial cellular dysfunction including ROS and apoptosis pathogenesis
PMID: 35468344
5
RARS2 phenotypic spectrum includes infantile myoclonic DEE; 85% of variants disrupt splicing or gene expression
PMID: 34717047
6
RARS2 variants cause PCH6 with presentations including hypoglycemia, lactic acidosis, and IESS
PMID: 38009286
7
RARS2 identified as mitochondrial disease gene in WES-based diagnosis of pediatric mitochondrial disorders
PMID: 27290639
8
RARS2 mutations cause mitochondrial translation disorders associated with epilepsy
PMID: 22283595
Disease Associationsβ“˜21
pontocerebellar hypoplasia type 6Open Targets
0.82Strong
pontocerebellar hypoplasiaOpen Targets
0.71Strong
Non-syndromic pontocerebellar hypoplasiaOpen Targets
0.53Moderate
genetic disorderOpen Targets
0.52Moderate
epilepsyOpen Targets
0.51Moderate
mitochondrial diseaseOpen Targets
0.44Moderate
isolated cerebellar hypoplasia/agenesisOpen Targets
0.32Weak
Loss of consciousnessOpen Targets
0.32Weak
mathematical abilityOpen Targets
0.29Weak
neurodegenerative diseaseOpen Targets
0.27Weak
Abnormal brain morphologyOpen Targets
0.27Weak
mineral metabolism diseaseOpen Targets
0.25Weak
smoking initiationOpen Targets
0.21Weak
atrial fibrillationOpen Targets
0.19Weak
asthmaOpen Targets
0.17Weak
gastroesophageal reflux diseaseOpen Targets
0.17Weak
Abnormality of the skeletal systemOpen Targets
0.16Weak
Severe intellectual disabilityOpen Targets
0.11Weak
benign urinary system neoplasmOpen Targets
0.04Suggestive
hypertrophic cardiomyopathyOpen Targets
0.03Suggestive
Pontocerebellar hypoplasia 6UniProt
Pathogenic Variants242
NM_020320.5(RARS2):c.1156C>T (p.Arg386Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 6|Inborn genetic diseases
β˜…β˜…β˜†β˜†2026β†’ Residue 386
NM_020320.5(RARS2):c.685C>T (p.Arg229Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2026β†’ Residue 229
NM_020320.5(RARS2):c.419T>G (p.Phe140Cys)Pathogenic
not provided|Inborn genetic diseases|Pontocerebellar hypoplasia type 6|Pontoneocerebellar hypoplasia
β˜…β˜…β˜†β˜†2026β†’ Residue 140
NM_020320.5(RARS2):c.1544A>G (p.Asp515Gly)Pathogenic
Pontocerebellar hypoplasia type 6|not provided|Pontoneocerebellar hypoplasia
β˜…β˜…β˜†β˜†2026β†’ Residue 515
NM_020320.5(RARS2):c.839T>G (p.Leu280Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2026β†’ Residue 280
NM_020320.5(RARS2):c.1A>G (p.Met1Val)Pathogenic
Inborn genetic diseases|not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2026β†’ Residue 1
NM_020320.5(RARS2):c.436_437delinsA (p.Arg146fs)Pathogenic
Pontocerebellar hypoplasia type 6|Pontoneocerebellar hypoplasia
β˜…β˜…β˜†β˜†2026β†’ Residue 146
NM_020320.5(RARS2):c.1554del (p.Arg519fs)Pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2026β†’ Residue 519
NM_020320.5(RARS2):c.297+2T>GPathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2026
NM_020320.5(RARS2):c.35A>G (p.Gln12Arg)Pathogenic
Pontocerebellar hypoplasia type 6|Inborn genetic diseases|not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 12
NM_020320.5(RARS2):c.45_46del (p.Arg15fs)Likely pathogenic
Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2026β†’ Residue 15
NM_020320.5(RARS2):c.16C>T (p.Arg6Cys)Likely pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2026β†’ Residue 6
NM_020320.5(RARS2):c.395+1G>ALikely pathogenic
Pontocerebellar hypoplasia type 6|not provided
β˜…β˜…β˜†β˜†2026
NM_020320.5(RARS2):c.1423G>T (p.Glu475Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2025β†’ Residue 475
NM_020320.5(RARS2):c.879-1G>CPathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2025
NM_020320.5(RARS2):c.1415+2T>CLikely pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2025
NM_020320.5(RARS2):c.127C>T (p.Gln43Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2025β†’ Residue 43
NM_020320.5(RARS2):c.1564G>A (p.Val522Ile)Pathogenic
Pontocerebellar hypoplasia type 6|not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 522
NM_020320.5(RARS2):c.2T>C (p.Met1Thr)Pathogenic
not provided|Pontocerebellar hypoplasia type 6
β˜…β˜…β˜†β˜†2025β†’ Residue 1
NM_020320.5(RARS2):c.110+5A>GPathogenic
Pontocerebellar hypoplasia type 6|not provided
β˜…β˜…β˜†β˜†2025
View on ClinVar β†—
Related Genes
TSEN34Protein interaction100%CARS1Protein interaction92%GARS1Protein interaction92%HARS1Protein interaction92%NARS1Protein interaction92%OATProtein interaction92%
Tissue Expression6 tissues
Brain
100%
Heart
90%
Lung
74%
Ovary
71%
Liver
71%
Bone Marrow
70%
Gene Interaction Network
Click a node to explore
RARS2TSEN34CARS1GARS1HARS1NARS1OAT
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q5T160
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
0.89LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.70 [0.56–0.89]
RankingsWhere RARS2 stands among ~20K protein-coding genes
  • #7,701of 20,598
    Most Researched60
  • #263of 5,498
    Most Pathogenic Variants242 Β· top 5%
  • #7,912of 17,882
    Most Constrained (LOEUF)0.89
Genes detectedRARS2
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
New perspective in diagnostics of mitochondrial disorders: two years' experience with whole-exome sequencing at a national paediatric centre.
PMID: 27290639
J Transl Med Β· 2016
1.00
2
Investigation in yeast of novel variants in mitochondrial aminoacyl-tRNA synthetases WARS2, NARS2, and RARS2 genes associated with mitochondrial diseases.
PMID: 39230874
Hum Mol Genet Β· 2024
0.90
3
Expanded clinical phenotype and untargeted metabolomics analysis in RARS2-related mitochondrial disorder: a case report.
PMID: 38438854
BMC Neurol Β· 2024
0.80
4
Infantile-onset myoclonic developmental and epileptic encephalopathy: A new RARS2 phenotype.
PMID: 34717047
Epilepsia Open Β· 2022
0.70
5
A non-coding variant in the Kozak sequence of RARS2 strongly decreases protein levels and causes pontocerebellar hypoplasia.
PMID: 37344844
BMC Med Genomics Β· 2023
0.60