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Gene Review

RPE65  -  retinal pigment epithelium-specific...

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Disease relevance of RPE65

  • The identification of RPE65 as the visual cycle target for the retinoic acids makes it feasible to develop useful drugs to treat retinal and macular degeneration while avoiding the substantial side effects of the retinoic acids [1].
  • It is shown here by using a quantitative fluorescence technique, that Accutane (13-cis-retinoic acid), a drug used in the treatment of acne but that causes night blindness, binds to RPE65 with a K(D) of 195 nM [1].
  • METHODS: Rats of four inbred strains (Lewis, Brown Norway, Fischer, and SHR) were immunized with native or recombinant bovine RPE65, or with S-antigen (S-Ag), emulsified with complete Freund adjuvant, and treated simultaneously with killed Bordetella pertussis bacteria, as indicated [2].

High impact information on RPE65


Biological context of RPE65

  • We compared the levels of translation products and mRNA stability among RPE65 transcripts containing deletions of the 5'- and 3'-UTRs [5].

Anatomical context of RPE65


Associations of RPE65 with chemical compounds


Analytical, diagnostic and therapeutic context of RPE65


  1. The specific binding of retinoic acid to RPE65 and approaches to the treatment of macular degeneration. Gollapalli, D.R., Rando, R.R. Proc. Natl. Acad. Sci. U.S.A. (2004) [Pubmed]
  2. RPE65 is highly uveitogenic in rats. Ham, D.I., Gentleman, S., Chan, C.C., McDowell, J.H., Redmond, T.M., Gery, I. Invest. Ophthalmol. Vis. Sci. (2002) [Pubmed]
  3. A palmitoylation switch mechanism in the regulation of the visual cycle. Xue, L., Gollapalli, D.R., Maiti, P., Jahng, W.J., Rando, R.R. Cell (2004) [Pubmed]
  4. Small molecule RPE65 antagonists limit the visual cycle and prevent lipofuscin formation. Maiti, P., Kong, J., Kim, S.R., Sparrow, J.R., Allikmets, R., Rando, R.R. Biochemistry (2006) [Pubmed]
  5. Role of the 3'-untranslated region of RPE65 mRNA in the translational regulation of the RPE65 gene: identification of a specific translation inhibitory element. Liu, S.Y., Redmond, T.M. Arch. Biochem. Biophys. (1998) [Pubmed]
  6. A cleavable affinity biotinylating agent reveals a retinoid binding role for RPE65. Jahng, W.J., David, C., Nesnas, N., Nakanishi, K., Rando, R.R. Biochemistry (2003) [Pubmed]
  7. Lack of effect of RPE65 removal on the enzymatic processing of all-trans-retinol into 11-cis-retinol in vitro. Choo, D.W., Cheung, E., Rando, R.R. FEBS Lett. (1998) [Pubmed]
  8. Identification of the RPE65 protein in mammalian cone photoreceptors. Znoiko, S.L., Crouch, R.K., Moiseyev, G., Ma, J.X. Invest. Ophthalmol. Vis. Sci. (2002) [Pubmed]
  9. RPE65 operates in the vertebrate visual cycle by stereospecifically binding all-trans-retinyl esters. Gollapalli, D.R., Maiti, P., Rando, R.R. Biochemistry (2003) [Pubmed]
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