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10 sources retrieved · Most recent: April 2026 · Index updated 14 days ago
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PDE6H
phosphodiesterase 6H
Chromosome 12 · 12p12.3
NCBI Gene: 5149Ensembl: ENSG00000139053.4HGNC: HGNC:8790UniProt: Q13956
24PubMed Papers
21Diseases
2Drugs
0Pathogenic Variants
CLINICAL
FDA Approved TargetOMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
protein bindingpositive regulation of epidermal growth factor receptor signaling pathwaypositive regulation of G protein-coupled receptor signaling pathwayphotoreceptor outer segment membraneachromatopsiaachromatopsia 6coronary artery diseasestroke
✦AI Summary

PDE6H encodes the gamma subunit of cone-specific cyclic GMP phosphodiesterase, a critical component of the phototransduction cascade in cone photoreceptors 1. PDE6H functions as a multisubunit enzyme that degrades cyclic GMP (cGMP) at specifically tuned rates, enabling signal amplification and transmission during visual perception 2. The protein is expressed specifically in cone outer segments and synaptic terminals, particularly in short and long/middle wavelength-sensitive cones 2. Pathogenic variants in PDE6H cause achromatopsia (color blindness), an autosomal recessive cone dysfunction syndrome affecting approximately 1 in 30,000 people 3. While CNGA3 and CNGB3 mutations account for up to 90% of achromatopsia cases, PDE6H mutations represent a significant genetic cause 3. PDE6H-associated achromatopsia presents at birth or early infancy with poor visual acuity, nystagmus, photophobia, and complete color vision loss, with variable cone subtype involvement 2. Notably, short-wave cone function is often more preserved than middle and long-wave cone function in PDE6H patients, suggesting differential cone vulnerability 2. Currently, no FDA-approved treatment exists, though multiple gene therapy approaches are under investigation in clinical trials 3. Additionally, PDE6H-derived promoters show promise for directing transgene expression in degenerating photoreceptors during advanced retinal disease stages 4.

Sources cited
1
PDE6H encodes the gamma subunit of human cone-specific cGMP phosphodiesterase located on chromosome 12p13
PMID: 8786098
2
PDE6H functions in degrading cGMP at specifically tuned rates in cone phototransduction and is expressed in cone outer segments and synaptic terminals
PMID: 25739440
3
PDE6H mutations cause achromatopsia, an autosomal recessive cone dysfunction syndrome affecting ~1 in 30,000 people with presentation of poor visual acuity, nystagmus, photophobia, and color vision loss
PMID: 39273686
4
PDE6H-associated achromatopsia shows variable cone subtype involvement with short-wave cones more preserved than middle and long-wave cones
PMID: 25739440
5
No FDA-approved treatment currently exists for achromatopsia, but multiple gene therapy clinical trials are registered at phase I/II stage
PMID: 39273686
6
PDE6H-derived promoters enable robust photoreceptor-specific transgene expression in late-stage retinal degeneration for AAV-mediated gene therapy
PMID: 40405464
Disease Associationsⓘ21
achromatopsiaOpen Targets
0.59Moderate
achromatopsia 6Open Targets
0.57Moderate
coronary artery diseaseOpen Targets
0.54Moderate
strokeOpen Targets
0.54Moderate
cone dystrophy 3Open Targets
0.40Weak
cardiovascular diseaseOpen Targets
0.39Weak
Retinal dystrophyOpen Targets
0.38Weak
cone dystrophyOpen Targets
0.37Weak
hypertensionOpen Targets
0.37Weak
intermittent vascular claudicationOpen Targets
0.37Weak
Recurrent thrombophlebitisOpen Targets
0.37Weak
chronic kidney diseaseOpen Targets
0.35Weak
Hepatitis, AlcoholicOpen Targets
0.33Weak
Neonatal sepsisOpen Targets
0.32Weak
non-alcoholic steatohepatitisOpen Targets
0.31Weak
COVID-19Open Targets
0.29Weak
necrotizing enterocolitisOpen Targets
0.28Weak
osteonecrosisOpen Targets
0.28Weak
chronic renal failure syndromeOpen Targets
0.27Weak
endometriosisOpen Targets
0.26Weak
Achromatopsia 6UniProt
Pathogenic Variants
No pathogenic variants reported on ClinVar for this gene.
View on ClinVar ↗
Drug Targets2
DIPYRIDAMOLEApproved
3',5'-cyclic phosphodiesterase inhibitor
coronary artery disease
PENTOXIFYLLINEApproved
Adenosine A2 receptor antagonist
cardiovascular disease
Related Genes
PDE6GShared pathway100%KCNV2Protein interaction98%APRTProtein interaction91%GMPSProtein interaction90%ITPAProtein interaction90%ARR3Protein interaction89%
Tissue Expression6 tissues
Bone Marrow
100%
Brain
20%
Lung
4%
Liver
0%
Heart
0%
Ovary
0%
Gene Interaction Network
Click a node to explore
PDE6HPDE6GKCNV2APRTGMPSITPAARR3
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted · UniProt Q13956
View on AlphaFold ↗
Constraintⓘ
LOEUFⓘ
1.90LoF Tolerant
pLIⓘ
0.01Tolerant
Observed/Expected LoF1.28 [0.63–1.90]
RankingsWhere PDE6H stands among ~20K protein-coding genes
  • #13,265of 20,598
    Most Researched24
  • #693of 1,025
    FDA-Approved Drug Targets2
  • #17,289of 17,882
    Most Constrained (LOEUF)1.90
Genes detectedPDE6H
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
Gene Therapy for Achromatopsia.
PMID: 39273686
Int J Mol Sci · 2024
1.00
2
Phenotyping and genotyping inherited retinal diseases: Molecular genetics, clinical and imaging features, and therapeutics of macular dystrophies, cone and cone-rod dystrophies, rod-cone dystrophies, Leber congenital amaurosis, and cone dysfunction syndromes.
PMID: 38278208
Prog Retin Eye Res · 2024
0.90
3
Monogenic Retinal Diseases Associated With Genes Encoding Phototransduction Proteins: A Review.
PMID: 40013354
Clin Exp Ophthalmol · 2025
0.80
4
Targeted ablation of the Pde6h gene in mice reveals cross-species differences in cone and rod phototransduction protein isoform inventory.
PMID: 25739440
J Biol Chem · 2015
0.70
5
Isolation and chromosomal localization of the human cone cGMP phosphodiesterase gamma cDNA (PDE6H).
PMID: 8786098
Genomics · 1996
0.60