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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.
DPH1
diphthamide biosynthesis 1
Chromosome 17 Β· 17p13.3
NCBI Gene: 1801Ensembl: ENSG00000108963.19HGNC: HGNC:3003UniProt: Q9BZG8
49PubMed Papers
21Diseases
0Drugs
14Pathogenic Variants
FUNCTIONAL ROLE
Tumor Suppressor
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
nucleoplasmprotein bindingprotein histidyl modification to diphthamide2-(3-amino-3-carboxypropyl)histidine synthase complexdevelopmental delay with short stature, dysmorphic facial features, and sparse hair 1developmental delay with short stature, dysmorphic facial features, and sparse hairneurodegenerative diseasegenetic disorder
✦AI Summary

DPH1 (diphthamide biosynthesis 1) catalyzes the first committed step of diphthamide biosynthesis, a post-translational modification of a histidine residue on elongation factor 2 (EEF2) 1. DPH1 and DPH2 work together to transfer a 3-amino-3-carboxypropyl group from S-adenosyl-L-methionine to this critical histidine, with assistance from DPH3 and NADH-dependent reductase components 1. Diphthamide modification is essential for proper translational fidelity and protein synthesis elongation in eukaryotic ribosomes 2. Autosomal-recessive DPH1 mutations cause diphthamide deficiency syndrome, characterized by intellectual disability, developmental delay, short stature, dysmorphic facial features, sparse hair, seizures, and craniofacial abnormalities 34. This phenotype results from impaired EEF2 function and compromised protein synthesis 4. Variant analysis has identified multiple loss-of-function alleles that reduce enzyme activity and catalytic efficiency 5. Notably, DPH1 downregulation in myeloid malignancies confers resistance to tagraxofusp (a diphtheria toxin-based therapeutic), though azacitidine can restore DPH1 expression and drug sensitivity 6. Additionally, DPH1 variants are implicated as risk factors in bipolar disorder based on fine-mapping analyses 7.

Sources cited
1
DPH1 catalyzes the first step of diphthamide biosynthesis on EEF2; supports functional analysis of DPH1 variants and their correlation with clinical severity
PMID: 30877278
2
Validates DPH1 as a disease gene for autosomal recessive intellectual disability with short stature, craniofacial, and ectodermal anomalies
PMID: 26220823
3
DPH1 defects impair EEF2 function and protein synthesis, resulting in developmental delay, short stature, and dysmorphic features
PMID: 29362492
4
Identifies loss-of-function DPH1 and DPH2 variants that cause diphthamide deficiency syndrome with intellectual disability and developmental abnormalities
PMID: 37675463
5
Diphthamide modification of eEF2 by DPH1 is conserved across eukaryotes and required for translational fidelity
PMID: 35817801
6
DPH1 downregulation confers tagraxofusp resistance in myeloid malignancies; azacitidine can restore DPH1 expression
PMID: 37846131
7
DPH1 identified as a bipolar disorder risk gene through fine-mapping genomic loci
PMID: 38405768
8
DPH1 initially identified as a novel disease gene in neurogenetic disorders via whole-exome sequencing
PMID: 25558065
Disease Associationsβ“˜21
developmental delay with short stature, dysmorphic facial features, and sparse hair 1Open Targets
0.77Strong
developmental delay with short stature, dysmorphic facial features, and sparse hairOpen Targets
0.67Moderate
neurodegenerative diseaseOpen Targets
0.49Moderate
genetic disorderOpen Targets
0.34Weak
intracerebral hemorrhageOpen Targets
0.27Weak
Global developmental delayOpen Targets
0.26Weak
Cerebellar vermis hypoplasiaOpen Targets
0.26Weak
Dandy-Walker syndromeOpen Targets
0.26Weak
HydrocephalusOpen Targets
0.26Weak
arthritisOpen Targets
0.14Weak
cervical cancerOpen Targets
0.07Suggestive
reticulum cell sarcomaOpen Targets
0.07Suggestive
Ewing sarcomaOpen Targets
0.06Suggestive
alveolar soft part sarcomaOpen Targets
0.06Suggestive
synovial sarcomaOpen Targets
0.06Suggestive
Robin sequence - oligodactylyOpen Targets
0.05Suggestive
Robin sequence-oligodactyly syndromeOpen Targets
0.05Suggestive
familial atypical multiple mole melanoma syndromeOpen Targets
0.05Suggestive
Orofaciodigital syndrome type 2Open Targets
0.05Suggestive
mesomelic dwarfism-cleft palate-camptodactyly syndromeOpen Targets
0.05Suggestive
Developmental delay with short stature, dysmorphic facial features, and sparse hair 1UniProt
Pathogenic Variants14
NM_001383.6(DPH1):c.274del (p.Glu92fs)Pathogenic
not provided|Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜…β˜…β˜†β˜†2024β†’ Residue 92
NM_001383.6(DPH1):c.686T>C (p.Leu229Pro)Pathogenic
Dandy-Walker syndrome;Cerebellar vermis hypoplasia;Hydrocephalus;Global developmental delay|Developmental delay with short stature, dysmorphic facial features, and sparse hair|Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜…β˜…β˜†β˜†2024β†’ Residue 229
NM_001383.6(DPH1):c.607dup (p.Cys203fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2025β†’ Residue 203
NM_001383.6(DPH1):c.400+1G>ALikely pathogenic
Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜…β˜†β˜†β˜†2025
NM_001383.6(DPH1):c.320A>G (p.Tyr107Cys)Pathogenic
not provided|Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜…β˜†β˜†β˜†2025β†’ Residue 107
NM_001383.6(DPH1):c.1007+1G>ALikely pathogenic
not provided
β˜…β˜†β˜†β˜†2025
NM_001383.6(DPH1):c.919C>T (p.Arg307Ter)Pathogenic
Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜…β˜†β˜†β˜†2024β†’ Residue 307
NM_001383.6(DPH1):c.103G>T (p.Glu35Ter)Pathogenic
Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜…β˜†β˜†β˜†2023β†’ Residue 35
NM_001383.6(DPH1):c.457del (p.Arg153fs)Likely pathogenic
Developmental delay with short stature, dysmorphic facial features, and sparse hair
β˜…β˜†β˜†β˜†2020β†’ Residue 153
NM_001383.6(DPH1):c.382T>C (p.Tyr128His)Likely pathogenic
Developmental delay with short stature, dysmorphic facial features, and sparse hair
β˜…β˜†β˜†β˜†2020β†’ Residue 128
NM_001383.6(DPH1):c.914_927del (p.Glu305fs)Pathogenic
not provided
β˜…β˜†β˜†β˜†2019β†’ Residue 305
NM_001383.6(DPH1):c.652C>T (p.Arg218Ter)Likely pathogenic
Developmental delay with short stature, dysmorphic facial features, and sparse hair 1|DPH1-related disorder
β˜…β˜†β˜†β˜†2019β†’ Residue 218
NM_001383.6(DPH1):c.476T>C (p.Leu159Pro)Pathogenic
Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜†β˜†β˜†β˜†2022β†’ Residue 159
NM_001383.6(DPH1):c.2T>A (p.Met1Lys)Pathogenic
not provided|Developmental delay with short stature, dysmorphic facial features, and sparse hair 1
β˜†β˜†β˜†β˜†2015β†’ Residue 1
View on ClinVar β†—
Related Genes
DPH5Protein interaction100%EEF2Protein interaction99%DPH6Protein interaction88%RBM8AProtein interaction88%DPH2Protein interaction84%DPH3Protein interaction81%
Tissue Expression6 tissues
Ovary
100%
Liver
91%
Bone Marrow
65%
Lung
61%
Heart
40%
Brain
21%
Gene Interaction Network
Click a node to explore
DPH1DPH5EEF2DPH6RBM8ADPH2DPH3
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q9BZG8
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
1.32LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF1.06 [0.86–1.32]
RankingsWhere DPH1 stands among ~20K protein-coding genes
  • #8,936of 20,598
    Most Researched49
  • #2,530of 5,498
    Most Pathogenic Variants14
  • #13,876of 17,882
    Most Constrained (LOEUF)1.32
Genes detectedDPH1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Accelerating novel candidate gene discovery in neurogenetic disorders via whole-exome sequencing of prescreened multiplex consanguineous families.
PMID: 25558065
Cell Rep Β· 2015
1.00
2
DPH1 and DPH2 variants that confer susceptibility to diphthamide deficiency syndrome in human cells and yeast models.
PMID: 37675463
Dis Model Mech Β· 2023
0.90
3
Novel compound heterozygous DPH1 mutations in a patient with the unique clinical features of airway obstruction and external genital abnormalities.
PMID: 29362492
J Hum Genet Β· 2018
0.80
4
Tagraxofusp in myeloid malignancies.
PMID: 37846131
Hematol Oncol Β· 2024
0.70
5
Matching two independent cohorts validates DPH1 as a gene responsible for autosomal recessive intellectual disability with short stature, craniofacial, and ectodermal anomalies.
PMID: 26220823
Hum Mutat Β· 2015
0.60