In pursuit of synthetic modulators for the orphan retina-specific nuclear receptor NR2E3

Journal of Ocular Pharmacology and Therapeutics

Volumn 29, Issue 3, 2013, Pages 298-309

  Qin, Qiong ^a   Knapinska, Anna ^b   Dobri, Nicoleta ^a   Madoux, Franck ^b   Chase, Peter ^b   Hodder, Peter ^b,c   Petrukhin, Konstantin ^a  

^a NewYork Presbyterian Hospital   (United States)

^b SCRIPPS RESEARCH INSTITUTE   (United States)

^c Scripps Florida ^*  (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AGENTS INTERACTING WITH TRANSMITTER, HORMONE OR DRUG RECEPTORS; COMPOUND 11A; COREPRESSOR PROTEIN; NR2E3 PROTEIN; NR2E3 PROTEIN AGONIST; NUCLEAR RECEPTOR COREPRESSOR; ORPHAN NUCLEAR RECEPTOR; PEROXISOME PROLIFERATOR ACTIVATED RECEPTOR GAMMA; RETCOR PROTEIN; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; DRUG SCREENING; DRUG SPECIFICITY; HIGH THROUGHPUT SCREENING; NONHUMAN; PROTEIN EXPRESSION; PROTEIN ISOLATION; PROTEIN PROTEIN INTERACTION; PROTEIN PURIFICATION; TIME RESOLVED FLUORESCENCE ENERGY TRANSFER; TITRIMETRY;

ANIMALS; CHO CELLS; CRICETINAE; CRICETULUS; DRUG DESIGN; DRUG INVERSE AGONISM; FLUORESCENCE RESONANCE ENERGY TRANSFER; HIGH-THROUGHPUT SCREENING ASSAYS; HUMANS; ORPHAN NUCLEAR RECEPTORS; REPRODUCIBILITY OF RESULTS; SF9 CELLS; SMALL MOLECULE LIBRARIES; SPODOPTERA;

EID: 84876807233     PISSN: 10807683     EISSN: 15577732     Source Type: Journal    
DOI: 10.1089/jop.2012.0135     Document Type: Article

References (34)

1. Petrukhin, K. New therapeutic targets in atrophic agerelated macular degeneration. Expert Opin. Ther. Targets. 11: 625-639, 2007. (Pubitemid 46744687)
2. Mangelsdorf, D.J., Thummel, C., Beato, M, Herrlich, P., Schutz, G., Umesono, K., Blumberg, B., Kastner, P., Mark, M., Chambon, P., and Evans, R.M. The nuclear receptor superfamily: the second decade. Cell. 83:835-839, 1995.
3. Chawla, A., Repa, J.J., Evans, R.M., and Mangelsdorf, D.J. Nuclear receptors and lipid physiology: opening the X-files. Science. 294:1866-1870, 2001. (Pubitemid 33101573)
4. Francis, G.A., Fayard, E., Picard, F., and Auwerx, J. Nuclear receptors and the control of metabolism. Annu. Rev. Physiol. 65:261-311, 2003. (Pubitemid 38249155)
5. Chatterjee, V.K. Resistance to thyroid hormone, and peroxisome- proliferator-activated receptor gamma resistance. Biochem. Soc. Trans. 29:227-231, 2001. (Pubitemid 32587510)
6. Kobayashi, M., Takezawa, S., Hara, K., Yu, R.T., Umesono, Y., Agata, K., Taniwaki, M., Yasuda, K., and Umesono, K. Identification of a photoreceptor cell-specific nuclear receptor. Proc. Natl. Acad. Sci. U S A. 96:4814-4819, 1999. (Pubitemid 29214494)
7. Chen, J., Rattner, A., and Nathans, J. The rod photoreceptor-specific nuclear receptor Nr2e3 represses transcription of multiple cone-specific genes. J. Neurosci. 25:118-129, 2005. (Pubitemid 40110772)
8. Akhmedov, N.B., Piriev, N.I., Chang, B., Rapoport, A.L., Hawes, N.L., Nishina, P.M., Nusinowitz, S., Heckenlively, J.R., Roderick, T.H., Kozak, C.A., Danciger, M., Davisson, M.T., and Farber, D.B. A deletion in a photoreceptor-specific nuclear receptor mRNA causes retinal degeneration in the rd7 mouse. Proc. Natl. Acad. Sci. U S A. 97:5551-5556, 2000. (Pubitemid 30313756)
9. Bumsted O'Brien, K.M., Cheng, H., Jiang, Y., Schulte, D., Swaroop, A., and Hendrickson, A.E. Expression of photoreceptor-specific nuclear receptor NR2E3 in rod photoreceptors of fetal human retina. Invest. Ophthalmol. Vis. Sci. 45:2807-2812, 2004. (Pubitemid 38998880)
10. Peng, G.H., Ahmad, O., Ahmad, F., Liu, J., and Chen, S. The photoreceptor-specific nuclear receptor Nr2e3 interacts with Crx and exerts opposing effects on the transcription of rod versus cone genes. Hum. Mol. Genet. 14:747-764, 2005. (Pubitemid 40403272)
11. Cheng, H., Khanna, H., Oh, E.C., Hicks, D., Mitton, K.P., and Swaroop, A. Photoreceptor-specific nuclear receptor NR2E3 functions as a transcriptional activator in rod photoreceptors. Hum. Mol. Genet. 13:1563-1575, 2004. (Pubitemid 39136713)
12. Haider, N.B., Jacobson, S.G., Cideciyan, A.V., Swiderski, R., Streb, L.M., Searby, C., Beck, G., Hockey, R., Hanna, D.B., Gorman, S., Duhl, D., Carmi, R., Bennett, J., Weleber, R.G., Fishman, G.A., Wright, A.F., Stone, E.M., and Sheffield, V.C. Mutation of a nuclear receptor gene, NR2E3, causes enhanced S cone syndrome, a disorder of retinal cell fate. Nat. Genet. 24:127-131, 2000. (Pubitemid 30094711)
13. Sharon, D., Sandberg, M.A., Caruso, R.C., Berson, E.L., and Dryja, T.P. Shared mutations in NR2E3 in enhanced S-cone syndrome, Goldmann-Favre syndrome, and many cases of clumped pigmentary retinal degeneration. Arch. Ophthalmol. 121:1316-1323, 2003. (Pubitemid 37128411)
14. Nakamura, M., Hotta, Y., Piao, C.H., Kondo, M., Terasaki, H., and Miyake, Y. Enhanced S-cone syndrome with subfoveal neovascularization. Am. J. Ophthalmol. 133:575-577, 2002. (Pubitemid 34258077)
15. Escher, P., Gouras, P., Roduit, R., Tiab, L., Bolay, S., Delarive, T., Chen, S., Tsai, C.-C., Hayashi, M., Zernant, J., Merriam, J.E., Mermod, N., Allikmets, R., Munier, F.L., and Schorderet, D.F. Mutations in NR2E3 can cause dominant or recessive retinal degenerations in the same family. Hum. Mutat. 30:342-351, 2009.
16. Coppieters, F., Leroy, B.P., Beysen, D., Hellemans, J., De Bosscher, K., Haegeman, G., Robberecht, K., Wuyts, W., Coucke, P.J., and De Baere, E. Recurrent mutation in the first zinc finger of the orphan nuclear receptor NR2E3 causes autosomal dominant retinitis pigmentosa. Am. J. Hum. Genet. 81:147-157, 2007. (Pubitemid 47001164)
17. Gerber, S., Rozet, J.M., Takezawa, S.I., dos Santos, L.C., Lopes, L., Gribouval, O., Penet, C., Perrault, I., Ducroq, D., Souied, E., Jeanpierre, M., Romana, S., Frezal, J., Ferraz, F., Yu-Umesono, R., Munnich, A., and Kaplan, J. The photore-ceptor cell-specific nuclear receptor gene (PNR) accounts for retinitis pigmentosa in the Crypto-Jews from Portugal (Marranos), survivors from the Spanish Inquisition. Hum. Genet. 107:276-284, 2000.
18. Haider, N.B., Naggert, J.K., and Nishina, P.M. Excess cone cell proliferation due to lack of a functional NR2E3 causes retinal dysplasia and degeneration in rd7/rd7 mice. Hum. Mol. Genet. 10:1619-1626, 2001. (Pubitemid 32776290)
19. Milam, A.H., Rose, L., Cideciyan, A.V., Barakat, M.R., Tang, W.X., Gupta, N., Aleman, T.S., Wright, A.F., Stone, E.M., Sheffield, V.C., and Jacobson, S.G. The nuclear receptor NR2E3 plays a role in human retinal photoreceptor differentiation and degeneration. Proc. Natl. Acad. Sci. U S A. 99:473-478, 2002. (Pubitemid 34060385)
20. Ueno, S., Kondo, M., Miyata, K., Hirai, T., Miyata, T., Usukura, J., Nishizawa, Y., and Miyake, Y. Physiological function of S-cone system is not enhanced in rd7 mice. Exp. Eye Res. 81:751-758, 2005. (Pubitemid 41790423)
21. Webber, A.L., Hodor, P., Thut, C.J., Vogt, T.F., Zhang, T., Holder, D.J., and Petrukhin, K. Dual role of Nr2e3 in photoreceptor development and maintenance. Exp. Eye Res. 87:35-48, 2008.
22. Kapitskaya, M., Cunningham, M.E., Lacson, R., Kornienko, O., Bednar, B., and Petrukhin, K. Development of the high throughput screening assay for identification of agonists of an orphan nuclear receptor. Assay Drug Dev. Technol. 4:253-262, 2006. (Pubitemid 44083580)
23. Wolkenberg, S.E., Zhao, Z., Kapitskaya, M., Webber, A.L., Petrukhin, K., Tang, Y.S., Dean, D.C., Hartman, G.D., and Lindsley, C.W. Identification of potent agonists of photore-ceptor-specific nuclear receptor (NR2E3) and preparation of a radioligand. Bioorg. Med. Chem. Lett. 16:5001-5004, 2006. (Pubitemid 44247714)
24. Chen, J.D., and Evans, R.M. A transcriptional co-repressor that interacts with nuclear hormone receptors. Nature. 377:454-457, 1995.
25. Horlein, A.J., Naar, A.M., Heinzel, T., Torchia, J., Gloss, B., Kurokawa, R., Ryan, A., Kamei, Y., Soderstrom, M., Glass, C.K., et al. Ligand-independent repression by the thyroid hormone receptor mediated by a nuclear receptor co-repressor. Nature. 377:397-404, 1995.
26. Takezawa, S., Yokoyama, A., Okada, M., Fujiki, R., Iriyama, A., Yanagi, Y., Ito, H., Takada, I., Kishimoto, M., Miyajima, A., Takeyama K.-I., Umesono, K., Kitagawa, H., and Kato, S. A cell cycle-dependent co-repressor mediates photoreceptor cell-specific nuclear receptor function. EMBO J. 26:764-774, 2007.
27. Latchman, D.S. Transcription Factors: A Practical Approach. United Kingdom: IRL; 1993.
28. Zhang, J.H., Chung, T.D., and Oldenburg, K.R. A Simple statistical parameter for use in evaluation and validation of high throughput screening assays. J. Biomol. Screen. 4:67-73, 1999. (Pubitemid 29278954)
29. Bondos, S.E., and Bicknell, A. Detection and prevention of protein aggregation before, during, and after purification. Anal. Biochem. 316:223-231, 2003. (Pubitemid 36444552)
30. Fujimura T, Sakuma H, Ohkubo-Suzuki A, Aramori I, Mutoh S. Unique properties of coactivator recruitment caused by differential binding of FK614, an anti-diabetic agent, to peroxisome proliferator-activated receptor gamma. Biol Pharm Bull. 29:423-429, 2006.
31. Hilal, T., Puetter, V., Otto, C., Parczyk, K., and Bader, B. A dual estrogen receptor TR-FRET assay for simultaneous measurement of steroid site binding and coactivator recruitment. J. Biomol. Screen. 15:268-278, 2010.
32. Chin, J., Adams, A.D., Bouffard, A., Green, A., Lacson, R.G., Smith, T., Fischer, P.A., Menke, J.G., Sparrow, C.P., and Mitnaul, L.J. Miniaturization of cell-based beta-lactamasedependent FRET assays to ultra-high throughput formats to identify agonists of human liver X receptors. Assay Drug Dev. Technol. 1:777-787, 2003.
33. Glickman, J.F., Wu, X., Mercuri, R., Illy, C., Bowen, B.R., He, Y., and Sills, M. A comparison of ALPHAScreen, TR-FRET, and TRF as assay methods for FXR nuclear receptors. J. Biomol. Screen. 7:3-10, 2002. (Pubitemid 34208857)
34. Madoux, F., Li, X., Chase, P., Zastrow, G., Cameron, M.D., Conkright, J.J., Griffin, P.R., Thacher, S., and Hodder, P. Potent, selective and cell penetrant inhibitors of SF-1 by functional ultra-high-throughput screening. Mol. Pharmacol. 73:1776-1784, 2008. (Pubitemid 351724365)