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Target intelligence / Profile preview
Retinal Pigment Epithelium Visual Cycle Enzyme (RPE65)
- Target
- RPE65
- Molecular classification
- Enzyme, Carotenoid cleavage oxygenase superfamily, Non-heme iron oxygenase
01
Overview
RPE65 is a critical enzyme in the visual cycle, specifically expressed in the retinal pigment epithelium (RPE) of the eye. It plays an essential role in regenerating 11-cis-retinal, which is necessary for both rod and cone-mediated vision. Mutations in RPE65 are linked to severe inherited retinal dystrophies, and gene therapy approaches have shown promising results in restoring some visual function in patients with LCA2 and related disorders.
- Other names
- Retinoid isomerohydrolase RPE65
02
Mechanism of action
Gene therapy to restore functional RPE65 expression
03
Biological functions
Visual phototransductionVitamin A/retinoid cycleRegeneration of 11-cis-retinal
04
Disease associations
Leber congenital amaurosis type 2 (LCA2)Autosomal recessive retinitis pigmentosa type 20
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