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GeneE
10 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.
TSEN54
tRNA splicing endonuclease subunit 54
Chromosome 17 Β· 17q25.1
NCBI Gene: 283989Ensembl: ENSG00000182173.15HGNC: HGNC:27561UniProt: Q7Z6J9
32PubMed Papers
23Diseases
0Drugs
84Pathogenic Variants
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
protein bindingtRNA-type intron splice site recognition and cleavagetRNA splicing, via endonucleolytic cleavage and ligationtRNA-intron endonuclease complexpontocerebellar hypoplasia type 4pontocerebellar hypoplasia type 2Apontocerebellar hypoplasia type 5pontocerebellar hypoplasia
✦AI Summary

TSEN54 encodes a non-catalytic subunit of the tRNA splicing endonuclease (TSEN) complex, which is essential for removing introns from precursor tRNAs 1. The complex recognizes the mature tRNA body structure and positions splice sites into catalytic centers for cleavage, releasing introns and two tRNA half-molecules with defined termini 2. TSEN54 cooperatively recognizes pre-tRNA through unique structural regions and helps trap the scissile phosphate for catalytic activity 2. Beyond tRNA processing, TSEN54 associates with pre-mRNA 3'-end processing factors, linking tRNA splicing to mRNA biogenesis. TSEN54 mutations cause pontocerebellar hypoplasia (PCH) types 2A, 4, and 5β€”rare autosomal recessive neurodegenerative disorders 3. Loss-of-function mechanisms underlie disease pathogenesis, with TSEN54 knockdown in zebrafish increasing brain cell death and causing structural abnormalities 3. Recent organoid models show PCH2a cerebellar organoids have reduced growth with altered proliferation kinetics 4. The cerebellum and pons show region-specific vulnerability, and prenatal MRI can differentiate PCH phenotypes based on vermis and cerebellar involvement patterns 5. Beyond neurological disease, TSEN54 upregulation in hepatocellular carcinoma associates with poor prognosis and altered immune cell infiltration 6.

Sources cited
1
TSEN54 is a subunit of human TSEN complex that recognizes mature tRNA domain and positions splice sites for catalytic cleavage
PMID: 37028420
2
TSEN54 cooperatively recognizes pre-tRNA through unique structural regions and participates in substrate positioning for both 3' and 5' cleavage
PMID: 37770519
3
TSEN54 mutations cause pontocerebellar hypoplasia through loss-of-function mechanism with increased brain cell death
PMID: 21273289
4
TSEN54-related PCH associates with three phenotypes (PCH2A, PCH4, PCH5) that can be differentiated by prenatal MRI patterns
PMID: 35962274
5
PCH2a cerebellar organoids with TSEN54 mutations show reduced size and altered proliferation kinetics in stem cell zones
PMID: 39034883
6
TSEN54 is upregulated in hepatocellular carcinoma and associated with poor prognosis and altered immune cell infiltration
PMID: 37059591
Disease Associationsβ“˜23
pontocerebellar hypoplasia type 4Open Targets
0.80Strong
pontocerebellar hypoplasia type 2AOpen Targets
0.78Strong
pontocerebellar hypoplasia type 5Open Targets
0.64Moderate
pontocerebellar hypoplasiaOpen Targets
0.62Moderate
Non-syndromic pontocerebellar hypoplasiaOpen Targets
0.53Moderate
Olivopontocerebellar hypoplasiaOpen Targets
0.51Moderate
genetic disorderOpen Targets
0.50Moderate
Global developmental delayOpen Targets
0.43Moderate
methylmalonic aciduria and homocystinuria type cblDOpen Targets
0.43Moderate
pontocerebellar hypoplasia type 2Open Targets
0.37Weak
microcephalyOpen Targets
0.37Weak
Abnormality of the nervous systemOpen Targets
0.34Weak
amblyopiaOpen Targets
0.34Weak
Huppke-Brendel syndromeOpen Targets
0.34Weak
HypertoniaOpen Targets
0.34Weak
Intellectual disabilityOpen Targets
0.34Weak
isolated cerebellar hypoplasia/agenesisOpen Targets
0.34Weak
hepatocellular carcinomaOpen Targets
0.07Suggestive
peripheral arterial diseaseOpen Targets
0.03Suggestive
diabetes mellitusOpen Targets
0.03Suggestive
Pontocerebellar hypoplasia 2AUniProt
Pontocerebellar hypoplasia 4UniProt
Pontocerebellar hypoplasia 5UniProt
Pathogenic Variants84
NM_207346.3(TSEN54):c.919G>T (p.Ala307Ser)Pathogenic
Pontocerebellar hypoplasia type 2A|not provided|Microcephaly;Global developmental delay|Olivopontocerebellar hypoplasia|Pontocerebellar hypoplasia type 4|Pontocerebellar hypoplasia type 5|Hypertonia;Amblyopia;Global developmental delay|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A|Congenital cerebellar hypoplasia|Inborn genetic diseases|Pontocerebellar hypoplasia type 2;Pontocerebellar hypoplasia type 4|Intellectual disability|Methylmalonic aciduria and homocystinuria type cblD;Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 5|TSEN54-related disorder|Pontoneocerebellar hypoplasia|TSEN54 Pontocerebellar Hypoplasia|Uterine corpus endometrial carcinoma|Sarcoma|Thyroid cancer, nonmedullary, 1|Cervical cancer|Huppke-Brendel syndrome|Melanoma|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5
β˜…β˜…β˜†β˜†2026β†’ Residue 307
NM_207346.3(TSEN54):c.670_671del (p.Lys224fs)Pathogenic
not provided|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5
β˜…β˜…β˜†β˜†2025β†’ Residue 224
NM_207346.3(TSEN54):c.789_798del (p.Leu264fs)Pathogenic
not provided|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5
β˜…β˜…β˜†β˜†2025β†’ Residue 264
NM_207346.3(TSEN54):c.1060_1078dup (p.Ala360fs)Pathogenic
not provided
β˜…β˜…β˜†β˜†2025β†’ Residue 360
NM_207346.3(TSEN54):c.1156C>T (p.Gln386Ter)Pathogenic
not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 386
NM_207346.3(TSEN54):c.775C>T (p.Gln259Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5
β˜…β˜…β˜†β˜†2024β†’ Residue 259
NM_207346.3(TSEN54):c.371G>T (p.Gly124Val)Pathogenic
Pontocerebellar hypoplasia type 2A|Pontoneocerebellar hypoplasia|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 124
NM_207346.3(TSEN54):c.846_856del (p.Ala284fs)Pathogenic
not provided|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5
β˜…β˜…β˜†β˜†2024β†’ Residue 284
NM_207346.3(TSEN54):c.1039A>T (p.Lys347Ter)Pathogenic
not provided|Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5;Pontocerebellar hypoplasia type 4
β˜…β˜…β˜†β˜†2024β†’ Residue 347
NM_207346.3(TSEN54):c.856_862dup (p.Val288fs)Pathogenic
Inborn genetic diseases|Pontoneocerebellar hypoplasia|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 288
NM_207346.3(TSEN54):c.505C>T (p.Arg169Ter)Pathogenic
not provided|Inborn genetic diseases|Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 5
β˜…β˜…β˜†β˜†2024β†’ Residue 169
NM_207346.3(TSEN54):c.190C>T (p.Gln64Ter)Pathogenic
not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 64
NM_207346.3(TSEN54):c.1220C>T (p.Pro407Leu)Likely pathogenic
not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 407
NM_207346.3(TSEN54):c.991del (p.Glu331fs)Pathogenic
not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 331
NM_207346.3(TSEN54):c.953del (p.Pro318fs)Pathogenic
Pontocerebellar hypoplasia type 4;Pontocerebellar hypoplasia type 5;Methylmalonic aciduria and homocystinuria type cblD|not provided
β˜…β˜…β˜†β˜†2024β†’ Residue 318
NM_207346.3(TSEN54):c.940del (p.Leu314fs)Pathogenic
Pontocerebellar hypoplasia type 2A;Pontocerebellar hypoplasia type 4|not provided
β˜…β˜…β˜†β˜†2023β†’ Residue 314
NM_207346.3(TSEN54):c.736C>T (p.Gln246Ter)Pathogenic
Pontocerebellar hypoplasia type 4|Pontocerebellar hypoplasia type 5|not provided
β˜…β˜…β˜†β˜†2023β†’ Residue 246
NM_207346.3(TSEN54):c.547C>T (p.Gln183Ter)Pathogenic
not provided
β˜…β˜…β˜†β˜†2023β†’ Residue 183
NM_207346.3(TSEN54):c.1335del (p.Leu446fs)Pathogenic
Olivopontocerebellar hypoplasia|Pontoneocerebellar hypoplasia
β˜…β˜…β˜†β˜†2019β†’ Residue 446
NM_207346.3(TSEN54):c.1313+1G>ALikely pathogenic
not provided
β˜…β˜†β˜†β˜†2026
View on ClinVar β†—
Related Genes
TSEN15Protein interaction100%CSTF2Protein interaction100%CPSF4Protein interaction100%CPSF1Protein interaction100%TSEN34Protein interaction84%VRK1Protein interaction78%
Tissue Expression6 tissues
Liver
100%
Lung
88%
Bone Marrow
80%
Ovary
63%
Brain
34%
Heart
23%
Gene Interaction Network
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TSEN54TSEN15CSTF2CPSF4CPSF1TSEN34VRK1
PROTEIN STRUCTURE
Preparing viewer…
PDB8HMZ Β· 2.90 Γ… Β· EM
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
1.26LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF1.00 [0.81–1.26]
RankingsWhere TSEN54 stands among ~20K protein-coding genes
  • #11,644of 20,598
    Most Researched32
  • #894of 5,498
    Most Pathogenic Variants84 Β· top quartile
  • #13,266of 17,882
    Most Constrained (LOEUF)1.26
Genes detectedTSEN54
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
TSEN54 Gene-Related Pontocerebellar-Hypoplasia and Role of Prenatal MR Imaging: Besides the Common Posterior Fossa Cystic Malformations.
PMID: 35962274
Cerebellum Β· 2023
1.00
2
Structural basis of pre-tRNA intron removal by human tRNA splicing endonuclease.
PMID: 37028420
Mol Cell Β· 2023
0.90
3
Identification of Alternative Variants and Insertion of the Novel Polymorphic
PMID: 28083540
Int J Genomics Β· 2016
0.80
4
Impairment of the tRNA-splicing endonuclease subunit 54 (tsen54) gene causes neurological abnormalities and larval death in zebrafish models of pontocerebellar hypoplasia.
PMID: 21273289
Hum Mol Genet Β· 2011
0.70
5
Comprehensive analysis reveals TSEN54 as a robust prognosis biomarker and promising immune-related therapeutic target for hepatocellular carcinoma.
PMID: 37059591
Aging (Albany NY) Β· 2023
0.60