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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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DHDDS
dehydrodolichyl diphosphate synthase subunit
Chromosome 1 Β· 1p36.11
NCBI Gene: 79947Ensembl: ENSG00000117682.19HGNC: HGNC:20603UniProt: Q86SQ9
38PubMed Papers
22Diseases
0Drugs
42Pathogenic Variants
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
ditrans,polycis-polyprenyl diphosphate synthase [(2E,6E)-farnesyl diphosphate specific] activityendoplasmic reticulum membraneprotein bindingdehydrodolichyl diphosphate synthase complexretinitis pigmentosa 59developmental delay and seizures with or without movement abnormalitiesretinitis pigmentosaneurodegenerative disease
✦AI Summary

DHDDS (dehydrodolichyl diphosphate synthase subunit) encodes the catalytic subunit of the dehydrodolichyl diphosphate synthase complex, which forms with NUS1 to catalyze dolichol biosynthesis 1. DHDDS functions as a cis-prenyltransferase that catalyzes chain elongation of farnesyl diphosphate through multiple condensations with isopentenyl pyrophosphate, producing dehydrodolichyl diphosphate, a precursor for dolichyl phosphate 1. This dolichol serves as a sugar carrier essential for N-linked protein glycosylation in the endoplasmic reticulum 1. DHDDS mutations cause two distinct phenotypes. Autosomal recessive variants cause retinitis pigmentosa 59 (RP59), characterized by inner retinal degeneration and photoreceptor-bipolar cell synaptic dysfunction 2. Heterozygous de novo variants cause a neurodevelopmental and neurodegenerative disorder presenting with developmental delay, epilepsy, and progressive movement disorders including myoclonus, ataxia, and dystonia 3. Disease mechanisms extend beyond glycosylation defects; DHDDS patient cells show Niemann-Pick C-like endolysosomal dysfunction with cholesterol and ganglioside GM1 accumulation, suggesting impaired lysosomal trafficking and lipid homeostasis 4. Miglustat, an approved NPC therapy, improves these cellular phenotypes, offering potential therapeutic options 4. Clinical presentation shows variable symptom onset from infancy to adulthood with no clear genotype-phenotype correlation 5.

Sources cited
1
DHDDS is a cis-prenyltransferase catalyzing chain elongation of farnesyl diphosphate to form dehydrodolichyl diphosphate, a precursor for dolichyl phosphate involved in N-linked protein glycosylation
PMID: 28167250
2
DHDDS mutations cause retinitis pigmentosa 59 through defective photoreceptor-bipolar cell synaptic transmission with bipolar and amacrine cell degeneration
PMID: 40574710
3
De novo DHDDS variants cause neurodevelopmental and neurodegenerative disorder with developmental delay, epilepsy, myoclonus, and ataxia; variants cluster around the active site affecting catalytic activity
PMID: 34382076
4
DHDDS patient cells exhibit Niemann-Pick C-like endolysosomal dysfunction with cholesterol and ganglioside GM1 accumulation; miglustat treatment improves these phenotypes
PMID: 40003936
5
Heterozygous DHDDS variants show variable age of symptom onset from infancy to adulthood with no correlation to specific variants or patient sex; autism spectrum disorder present in 19% of patients
PMID: 39540616
Disease Associationsβ“˜22
retinitis pigmentosa 59Open Targets
0.78Strong
developmental delay and seizures with or without movement abnormalitiesOpen Targets
0.75Strong
retinitis pigmentosaOpen Targets
0.64Moderate
neurodegenerative diseaseOpen Targets
0.53Moderate
genetic disorderOpen Targets
0.47Moderate
congenital disorder of glycosylation, type IbbOpen Targets
0.42Moderate
DHDDS-CDGOpen Targets
0.40Weak
Retinal dystrophyOpen Targets
0.37Weak
undetermined early-onset epileptic encephalopathyOpen Targets
0.37Weak
HypercholesterolemiaOpen Targets
0.18Weak
Posterior column ataxia - retinitis pigmentosaOpen Targets
0.18Weak
upper respiratory tract disorderOpen Targets
0.15Weak
metabolic diseaseOpen Targets
0.14Weak
hyperlipidemiaOpen Targets
0.14Weak
coronary artery diseaseOpen Targets
0.12Weak
coronary atherosclerosisOpen Targets
0.11Weak
response to statinOpen Targets
0.09Suggestive
age-related macular degenerationOpen Targets
0.09Suggestive
Abnormality of the skeletal systemOpen Targets
0.09Suggestive
X-linked retinal dysplasiaOpen Targets
0.08Suggestive
Developmental delay and seizures with or without movement abnormalitiesUniProt
Retinitis pigmentosa 59UniProt
Pathogenic Variants42
NM_205861.3(DHDDS):c.124A>G (p.Lys42Glu)Pathogenic
Retinitis pigmentosa 59|Retinitis pigmentosa 59;Developmental delay and seizures with or without movement abnormalities|Retinitis pigmentosa|not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 42
NM_205861.3(DHDDS):c.109C>T (p.Arg37Cys)Pathogenic
not provided|Retinitis pigmentosa 59|Developmental delay and seizures with or without movement abnormalities|Retinitis pigmentosa 59;Developmental delay and seizures with or without movement abnormalities
β˜…β˜…β˜†β˜†2025β†’ Residue 37
NM_205861.3(DHDDS):c.632G>A (p.Arg211Gln)Pathogenic
Developmental delay and seizures with or without movement abnormalities|not provided|Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2025β†’ Residue 211
NM_205861.3(DHDDS):c.113G>A (p.Arg38His)Pathogenic
Developmental delay and seizures with or without movement abnormalities|Inborn genetic diseases
β˜…β˜…β˜†β˜†2025β†’ Residue 38
NM_205861.3(DHDDS):c.104G>A (p.Gly35Glu)Pathogenic
Inborn genetic diseases|not provided|Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2025β†’ Residue 35
NM_205861.3(DHDDS):c.109C>A (p.Arg37Ser)Pathogenic
not provided|Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2025β†’ Residue 37
NM_205861.3(DHDDS):c.658-2A>GLikely pathogenic
Retinitis pigmentosa 59|Retinitis pigmentosa 59;Developmental delay and seizures with or without movement abnormalities
β˜…β˜…β˜†β˜†2025
NM_205861.3(DHDDS):c.110G>A (p.Arg37His)Pathogenic
not provided|Developmental delay and seizures with or without movement abnormalities|Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2025β†’ Residue 37
NM_205861.3(DHDDS):c.517dup (p.Val173fs)Pathogenic
Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2025β†’ Residue 173
NM_205861.3(DHDDS):c.441-24A>GLikely pathogenic
Congenital disorder of glycosylation, type Ibb|Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2025
NM_205861.3(DHDDS):c.614G>A (p.Arg205Gln)Pathogenic
Retinitis pigmentosa 59|not provided|Developmental delay and seizures with or without movement abnormalities
β˜…β˜…β˜†β˜†2025β†’ Residue 205
NM_205861.3(DHDDS):c.616A>G (p.Thr206Ala)Likely pathogenic
Retinitis pigmentosa|Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2024β†’ Residue 206
NM_205861.3(DHDDS):c.665_668del (p.His222fs)Likely pathogenic
Retinitis pigmentosa 59|Retinitis pigmentosa 59;Developmental delay and seizures with or without movement abnormalities
β˜…β˜…β˜†β˜†2024β†’ Residue 222
NM_205861.3(DHDDS):c.698C>G (p.Pro233Arg)Pathogenic
Developmental delay and seizures with or without movement abnormalities|Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2024β†’ Residue 233
NM_205861.3(DHDDS):c.264_267del (p.Ser88fs)Pathogenic
Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2023β†’ Residue 88
NM_205861.3(DHDDS):c.513G>A (p.Trp171Ter)Pathogenic
Retinitis pigmentosa 59
β˜…β˜…β˜†β˜†2023β†’ Residue 171
NM_205861.3(DHDDS):c.820C>T (p.Gln274Ter)Likely pathogenic
Retinitis pigmentosa 59
β˜…β˜†β˜†β˜†2026β†’ Residue 274
NM_205861.3(DHDDS):c.827_828del (p.Thr276fs)Likely pathogenic
Retinitis pigmentosa 59
β˜…β˜†β˜†β˜†2025β†’ Residue 276
NM_205861.3(DHDDS):c.517del (p.Val173fs)Pathogenic
Retinitis pigmentosa 59
β˜…β˜†β˜†β˜†2025β†’ Residue 173
NM_205861.3(DHDDS):c.512G>A (p.Trp171Ter)Likely pathogenic
Retinitis pigmentosa 59
β˜…β˜†β˜†β˜†2025β†’ Residue 171
View on ClinVar β†—
Related Genes
FDFT1Protein interaction95%FDPSProtein interaction95%FNTAProtein interaction95%FNTBProtein interaction95%GGPS1Protein interaction95%PDSS1Protein interaction95%
Tissue Expression6 tissues
Heart
100%
Liver
96%
Lung
78%
Ovary
66%
Brain
62%
Bone Marrow
61%
Gene Interaction Network
Click a node to explore
DHDDSFDFT1FDPSFNTAFNTBGGPS1PDSS1
PROTEIN STRUCTURE
Preparing viewer…
PDB7PAX Β· 2.00 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
0.54Moderately Constrained
pLIβ“˜
0.91Intolerant
Observed/Expected LoF0.35 [0.24–0.54]
RankingsWhere DHDDS stands among ~20K protein-coding genes
  • #10,457of 20,598
    Most Researched38
  • #1,498of 5,498
    Most Pathogenic Variants42
  • #3,372of 17,882
    Most Constrained (LOEUF)0.54 Β· top quartile
Genes detectedDHDDS
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
PMID: 20301590
1.00
2
High Rate of Recurrent De Novo Mutations in Developmental and Epileptic Encephalopathies.
PMID: 29100083
Am J Hum Genet Β· 2017
0.90
3
DHDDS and NUS1: A Converging Pathway and Common Phenotype.
PMID: 38291835
Mov Disord Clin Pract Β· 2024
0.80
4
De novo DHDDS variants cause a neurodevelopmental and neurodegenerative disorder with myoclonus.
PMID: 34382076
Brain Β· 2022
0.70
5
Dhdds T206A and Dhdds K42E knock-in mouse models of retinitis pigmentosa 59 are phenotypically similar.
PMID: 40574710
Dis Model Mech Β· 2025
0.60