HomeAboutRankingsData Sources
Β© 2026 GeneE
🧬
GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 15 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
PUS3
pseudouridine synthase 3
Chromosome 11 Β· 11q24.2
NCBI Gene: 83480Ensembl: ENSG00000110060.10HGNC: HGNC:25461UniProt: A0A087WY59
18PubMed Papers
21Diseases
0Drugs
12Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
tRNA pseudouridine(38/39) synthase activitypseudouridine synthase activitytRNA pseudouridine synthesismRNA pseudouridine synthesissevere growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndromeHydrolethalushydrolethalus syndromeIntellectual disability
✦AI Summary

PUS3 (pseudouridine synthase 3) is a tRNA-modifying enzyme that catalyzes pseudouridylation at positions 38/39 in the anticodon stem-loop of transfer RNAs 1. The enzyme functions as a symmetric homodimer that recognizes tRNA substrates and positions target uridine residues for isomerization 1. Biochemical studies confirm PUS3 specifically modifies tRNAs but not mRNAs in human cells 1. Biallelic PUS3 variants cause rare neurodevelopmental disorders characterized by global developmental delay, epilepsy, hypotonia, and microcephaly 2. Disease-causing mutations impair protein thermostability or cause aggregation, leading to reduced PUS3 protein levels and diminished pseudouridine modifications in patient-derived cells 3. The clinical spectrum extends to include retinal dystrophy, cerebellar hypoplasia, and cardiac defects in severe cases 4. Recent evidence suggests PUS3 dysregulation participates in sepsis pathogenesis, with elevated PUS3 expression identified as a diagnostic biomarker (AUC 0.834) in septic patients 5. The connection between impaired tRNA modification and intellectual disability demonstrates that accurate tRNA biogenesis is essential for normal brain development.

Sources cited
1
PUS3 catalyzes pseudouridylation at positions 38/39 in tRNA anticodon stem-loop, functions as a homodimer, and does not modify mRNAs in human cells
PMID: 38996458
2
Biallelic PUS3 variants cause neurodevelopmental disorder dominated by global developmental delay, epilepsy, hypotonia, and microcephaly
PMID: 34415064
3
Disease-causing PUS3 mutations impair protein thermostability or cause aggregation, reducing PUS3 protein levels and pseudouridine modifications in patient cells
PMID: 36125428
4
PUS3-related disorders present with severe syndromic features including retinal dystrophy, cerebellar hypoplasia, and congenital heart defects
PMID: 34713961
5
PUS3 is dysregulated in sepsis and functions as a diagnostic biomarker with AUC of 0.834
PMID: 37701433
Disease Associationsβ“˜21
severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndromeOpen Targets
0.73Strong
HydrolethalusOpen Targets
0.50Moderate
hydrolethalus syndromeOpen Targets
0.45Moderate
Intellectual disabilityOpen Targets
0.37Weak
microcephalyOpen Targets
0.37Weak
Global developmental delayOpen Targets
0.37Weak
neurodegenerative diseaseOpen Targets
0.37Weak
genetic disorderOpen Targets
0.34Weak
Abnormal heart morphologyOpen Targets
0.33Weak
anencephalyOpen Targets
0.33Weak
Ankle flexion contractureOpen Targets
0.33Weak
Aplasia/Hypoplasia of the cerebellumOpen Targets
0.33Weak
congenital heart diseaseOpen Targets
0.33Weak
Isolated anencephaly/exencephalyOpen Targets
0.33Weak
PolyhydramniosOpen Targets
0.33Weak
Dandy-Walker syndromeOpen Targets
0.32Weak
Genetic syndrome with a Dandy-Walker malformation as major featureOpen Targets
0.32Weak
alcohol drinkingOpen Targets
0.03Suggestive
Neurodevelopmental disorderOpen Targets
0.01Suggestive
triple-negative breast cancerOpen Targets
0.01Suggestive
Neurodevelopmental disorder with microcephaly and gray scleraeUniProt
Pathogenic Variants12
NM_031307.4(PUS3):c.212A>G (p.Tyr71Cys)Pathogenic
not provided|Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 71
NM_031307.4(PUS3):c.838C>T (p.Arg280Ter)Pathogenic
Anencephaly;Heart, malformation of;Ankle flexion contracture;Aplasia/Hypoplasia of the cerebellum;Polyhydramnios|PUS3-related disorder|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 280
NM_031307.4(PUS3):c.1303C>T (p.Arg435Ter)Pathogenic
Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜…β˜†β˜†2024β†’ Residue 435
NM_031307.4(PUS3):c.366_367del (p.Ala123fs)Pathogenic
Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜…β˜†β˜†2022β†’ Residue 123
NM_031307.4(PUS3):c.78_79del (p.Arg27fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2024β†’ Residue 27
NM_031307.4(PUS3):c.438_442del (p.Asn146fs)Pathogenic
Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜†β˜†β˜†2023β†’ Residue 146
NM_031307.4(PUS3):c.993del (p.Asn331fs)Likely pathogenic
Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜†β˜†β˜†2022β†’ Residue 331
NM_031307.4(PUS3):c.17_18del (p.Thr6fs)Likely pathogenic
Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜†β˜†β˜†2021β†’ Residue 6
NM_031307.4(PUS3):c.981dup (p.Lys328Ter)Likely pathogenic
Inborn genetic diseases
β˜…β˜†β˜†β˜†2021β†’ Residue 328
NM_031307.4(PUS3):c.340T>C (p.Cys114Arg)Likely pathogenic
Anencephaly;Heart, malformation of;Ankle flexion contracture;Aplasia/Hypoplasia of the cerebellum;Polyhydramnios
β˜…β˜†β˜†β˜†2019β†’ Residue 114
NM_031307.4(PUS3):c.1048C>T (p.Gln350Ter)Likely pathogenic
Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜†β˜†β˜†2019β†’ Residue 350
NM_031307.4(PUS3):c.1181_1182del (p.Ser394fs)Likely pathogenic
Severe growth deficiency-strabismus-extensive dermal melanocytosis-intellectual disability syndrome
β˜…β˜†β˜†β˜†β†’ Residue 394
View on ClinVar β†—
Related Genes
MRPL17Protein interaction94%TRUB1Protein interaction88%WDR4Protein interaction81%PUSL1Shared pathway80%PUS7Protein interaction77%PUS7LProtein interaction76%
Tissue Expression6 tissues
Liver
100%
Brain
43%
Heart
32%
Lung
30%
Ovary
27%
Bone Marrow
10%
Gene Interaction Network
Click a node to explore
PUS3MRPL17TRUB1WDR4PUSL1PUS7PUS7L
PROTEIN STRUCTURE
Preparing viewer…
PDB9ENB Β· 2.66 Γ… Β· EM
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
1.06LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.80 [0.61–1.06]
RankingsWhere PUS3 stands among ~20K protein-coding genes
  • #14,786of 20,598
    Most Researched18
  • #2,717of 5,498
    Most Pathogenic Variants12
  • #10,629of 17,882
    Most Constrained (LOEUF)1.06
Genes detectedPUS3
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Identification and validation of key biomarkers based on RNA methylation genes in sepsis.
PMID: 37701433
Front Immunol Β· 2023
1.00
2
The molecular basis of tRNA selectivity by human pseudouridine synthase 3.
PMID: 38996458
Mol Cell Β· 2024
0.90
3
Destabilization of mutated human PUS3 protein causes intellectual disability.
PMID: 36125428
Hum Mutat Β· 2022
0.80
4
Clinical and molecular delineation of PUS3-associated neurodevelopmental disorders.
PMID: 34415064
Clin Genet Β· 2021
0.70
5
PUS3-related disorder: Report of a novel patient and delineation of the phenotypic spectrum.
PMID: 34713961
Am J Med Genet A Β· 2022
0.60