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GeneE
17 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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PTCD3
pentatricopeptide repeat domain 3
Chromosome 2 Β· 2p11.2
NCBI Gene: 55037Ensembl: ENSG00000132300.20HGNC: HGNC:24717UniProt: Q96EY7
156PubMed Papers
21Diseases
0Drugs
10Pathogenic Variants
FUNCTIONAL ROLE
Hub Gene
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
RNA bindingprotein bindingrRNA bindingribosomal small subunit bindingcombined oxidative phosphorylation deficiency 51mixed connective tissue diseasegenetic disorderalcohol drinking
✦AI Summary

PTCD3 (pentatricopeptide repeat domain 3) is a mitochondrial RNA-binding protein that associates with the small subunit of the mitochondrial ribosome and regulates mitochondrial translation 1. Rather than affecting mRNA stability or processing, PTCD3 promotes mitochondrial protein synthesis and respiratory chain function 1. Loss of PTCD3 function impairs the activity of oxidative phosphorylation complexes I and IV, resulting in decreased mitochondrial respiration and ATP biosynthesis 2. PTCD3 deficiency causes Leigh syndrome, a severe neurological disorder presenting with psychomotor delay, respiratory insufficiency, dystonia, optic atrophy, and seizures in early infancy 32. Brain imaging shows characteristic thalamic and basal ganglia abnormalities. Pathogenic variants identified include compound heterozygous mutations affecting mRNA processing and protein truncation, with functional studies demonstrating impaired mitochondrial translation machinery function 3. Beyond inherited disease, PTCD3 has been implicated in cancer progression. PTCD3 is upregulated in colorectal and prostate cancers, where it correlates with tumor stage, metastatic potential, and poor prognosis 45. In colorectal cancer, PTCD3 promotes glutaminolysis through IGF2BP2-mediated stabilization of SLC38A2 mRNA, supporting metabolic reprogramming that facilitates cancer cell growth and metastasis 5.

Sources cited
1
PTCD3 associates with mitochondrial small ribosomal subunit and regulates mitochondrial translation without affecting mRNA stability
PMID: 19427859
2
PTCD3 mutations cause oxidative phosphorylation defects, decreased complexes I and IV levels/activities, and Leigh syndrome
PMID: 30607703
3
PTCD3 deficiency causes Leigh syndrome with neurological symptoms and mitochondrial translation defects confirmed by functional complementation
PMID: 36450274
4
PTCD3 expression is elevated in prostate cancer and correlates with tumor progression and poor prognosis
PMID: 30132530
5
PTCD3 promotes colorectal cancer glutaminolysis and metastasis via IGF2BP2-mediated SLC38A2 mRNA stability
PMID: 40304977
Disease Associationsβ“˜21
combined oxidative phosphorylation deficiency 51Open Targets
0.67Moderate
mixed connective tissue diseaseOpen Targets
0.28Weak
genetic disorderOpen Targets
0.19Weak
alcohol drinkingOpen Targets
0.12Weak
posterior cortical atrophyOpen Targets
0.07Suggestive
multinodular goiterOpen Targets
0.06Suggestive
benign prostatic hyperplasiaOpen Targets
0.05Suggestive
hemoglobin D diseaseOpen Targets
0.05Suggestive
Hemoglobin E - beta-thalassemiaOpen Targets
0.05Suggestive
hemoglobin E-beta-thalassemia syndromeOpen Targets
0.05Suggestive
hereditary persistence of fetal hemoglobin-sickle cell disease syndromeOpen Targets
0.04Suggestive
dominant beta-thalassemiaOpen Targets
0.04Suggestive
delta-beta-thalassemiaOpen Targets
0.04Suggestive
hemoglobin E diseaseOpen Targets
0.04Suggestive
Hemoglobin C - beta-thalassemiaOpen Targets
0.04Suggestive
hemoglobin C-beta-thalassemia syndromeOpen Targets
0.04Suggestive
Alpha-thalassemia - myelodysplastic syndromeOpen Targets
0.04Suggestive
alpha-thalassemia-myelodysplastic syndromeOpen Targets
0.04Suggestive
hemoglobin H diseaseOpen Targets
0.04Suggestive
Hereditary persistence of fetal hemoglobin - beta-thalassemiaOpen Targets
0.03Suggestive
Combined oxidative phosphorylation deficiency 51UniProt
Pathogenic Variants10
NM_017952.6(PTCD3):c.1630-1G>ALikely pathogenic
Combined oxidative phosphorylation deficiency 51
β˜…β˜†β˜†β˜†2026
NM_017952.6(PTCD3):c.1979+1G>ALikely pathogenic
Combined oxidative phosphorylation deficiency 51
β˜…β˜†β˜†β˜†2024
NM_017952.6(PTCD3):c.1431G>A (p.Trp477Ter)Likely pathogenic
Combined oxidative phosphorylation deficiency 51
β˜…β˜†β˜†β˜†2024β†’ Residue 477
NM_017952.6(PTCD3):c.1148-2A>GLikely pathogenic
PTCD3-related disorder
β˜…β˜†β˜†β˜†2022
NM_017952.6(PTCD3):c.710del (p.Thr237fs)Likely pathogenic
See cases|Combined oxidative phosphorylation deficiency 51
β˜…β˜†β˜†β˜†2021β†’ Residue 237
NM_017952.6(PTCD3):c.1166C>G (p.Ser389Ter)Likely pathogenic
PTCD3-related disorder
β˜†β˜†β˜†β˜†2024β†’ Residue 389
NM_017952.6(PTCD3):c.415-2A>GPathogenic
Combined oxidative phosphorylation deficiency 51
β˜†β˜†β˜†β˜†2024
NM_017952.6(PTCD3):c.1746_1747dup (p.Phe583fs)Pathogenic
Combined oxidative phosphorylation deficiency 51
β˜†β˜†β˜†β˜†2024β†’ Residue 583
NM_017952.6(PTCD3):c.1453-1G>CPathogenic
Combined oxidative phosphorylation deficiency 51
β˜†β˜†β˜†β˜†2024
NM_017952.6(PTCD3):c.902C>T (p.Thr301Ile)Pathogenic
Combined oxidative phosphorylation deficiency 51
β˜†β˜†β˜†β˜†2024β†’ Residue 301
View on ClinVar β†—
Related Genes
MRPL54Shared pathway100%MRPL53Shared pathway100%MRPL57Shared pathway100%MRPL38Shared pathway100%MRPS6Protein interaction100%MRPS9Protein interaction100%
Tissue Expression6 tissues
Heart
100%
Bone Marrow
65%
Liver
52%
Ovary
51%
Brain
35%
Lung
34%
Gene Interaction Network
Click a node to explore
PTCD3MRPL54MRPL53MRPL57MRPL38MRPS6MRPS9
PROTEIN STRUCTURE
Preparing viewer…
PDB7QI4 Β· 2.21 Γ… Β· EM
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
1.13LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.92 [0.75–1.13]
RankingsWhere PTCD3 stands among ~20K protein-coding genes
  • #2,896of 20,598
    Most Researched156 Β· top quartile
  • #2,835of 5,498
    Most Pathogenic Variants10
  • #11,696of 17,882
    Most Constrained (LOEUF)1.13
Genes detectedPTCD3
Sources retrieved17 papers
Response timeβ€”
πŸ“„ Sources
17β–Ό
1
Epigenetic Activation of PTCD3 Promotes CRC Glutamine Metabolism and Metastasis via IGF2BP2-Mediated SLC38A2 m6A Modification.
PMID: 40304977
FASEB J Β· 2025
1.00
2
Leigh syndrome is the main clinical characteristic of PTCD3 deficiency.
PMID: 36450274
Brain Pathol Β· 2023
0.90
3
The role of the protein-RNA recognition code in neurodegeneration.
PMID: 30980111
Cell Mol Life Sci Β· 2019
0.80
4
Elevated expression of PTCD3 correlates with tumor progression and predicts poor prognosis in patients with prostate cancer.
PMID: 30132530
Mol Med Rep Β· 2018
0.70
5
Pentatricopeptide repeat domain protein 3 associates with the mitochondrial small ribosomal subunit and regulates translation.
PMID: 19427859
FEBS Lett Β· 2009
0.60