RPE65

Retinal pigment epithelium 65 kDa protein

RPE65 (Retinal pigment epithelium 65 kDa protein) is a critical enzyme in the visual cycle that is specifically expressed in retinal pigment epithelial (RPE) cells. As a retinoid isomerohydrolase, RPE65 catalyzes the conversion of all-trans-retinyl esters to 11-cis-retinol, a vital process for visual phototransduction ^[1].RPE65 facilitates retinoid isomerization through its iron-binding site, maintaining the continuous supply of 11-cis-retinal in the retina - the essential precursor for rhodopsin regeneration ^[2]. Deficiencies in RPE65 function disrupt the visual cycle and are associated with various inherited retinal disorders, including Leber congenital amaurosis type 2 (LCA2) and retinitis pigmentosa ^[3].

参考文献:

[1]. Gu SM, et al. Mutations in RPE65 cause autosomal recessive childhood-onset severe retinal dystrophy. Nat Genet. 1997 Oct;17(2):194-7. [Content Brief]

[2]. Moiseyev G, et al. RPE65 is the isomerohydrolase in the retinoid visual cycle. Proc Natl Acad Sci U S A. 2005 Aug 30;102(35):12413-8. [Content Brief]

[3]. Russell S, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017 Aug 26;390(10097):849-860. [Content Brief]

RPE65 相关产品 (1):

Cat. No.	Product Name	Effect	Purity	Chemical Structure

HY-19720

Emixustat		98.00%
Emixustat 是具口服活性的 RPE65 抑制剂，IC₅₀ 值为 4.4 nM。Emixustat 也是视觉周期调节剂，可通过抑制视黄醇异构化来调节视觉周期活动，有望用于研究视力疾病，如年龄相关性黄斑变性 (AMD)。

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