Therapeutic rescue of misfolded/mistrafficked mutants: Automation-friendly high-throughput assays for identification of pharmacoperone drugs of gpcrs

Methods in Enzymology

Volumn 521, Issue , 2013, Pages 3-16

  Smithson, David C ^a   Janovick, Jo Ann ^b   Conn, P Michael ^a,b,c  

^a Drug Development Institute   (United States)

^b OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^c OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

Author keywords

G protein coupled receptors; GPCR trafficking; GPCRs; High throughput screen; HTS; Misfolded GPCRs; Mutant rescue; Pharmacoperones; Quality control systems

Indexed keywords

CHAPERONE; G PROTEIN COUPLED RECEPTOR; GONADORELIN RECEPTOR; PHARMACOPERONE; SATAVAPTAN; UNCLASSIFIED DRUG; VASOPRESSIN V2 RECEPTOR;

ARTICLE; AUTOMATION; DATA ANALYSIS; DRUG DETERMINATION; DRUG IDENTIFICATION; HIGH THROUGHPUT SCREENING; HUMAN; HUMAN CELL; MUTATION; PRIORITY JOURNAL; PROTEIN CONFORMATION; PROTEIN EXPRESSION; PROTEIN FOLDING; ROBOTICS;

EID: 84873054348     PISSN: 00766879     EISSN: 15577988     Source Type: Book Series    
DOI: 10.1016/B978-0-12-391862-8.00001-6     Document Type: Chapter

References (42)

1. M.D. Amaral Therapy through chaperones: Sense or antisense? Cystic fibrosis as a model disease Journal of Inherited Metabolic Disease 29 2006 477 487
2. G.B. Benedek, J. Pande, G.M. Thurston, and J.I. Clark Theoretical and experimental basis for the inhibition of cataract Progress in Retinal and Eye Research 18 1999 391 402
3. V. Bernier, D.G. Bichet, and M. Bouvier Pharmacological chaperone action on G-protein-coupled receptors Current Opinion in Pharmacology 4 2004 528 533
4. V. Bernier, M. Lagace, D.G. Bichet, and M. Bouvier Pharmacological chaperones: Potential treatment for conformational diseases Trends in Endocrinology and Metabolism 15 2004 222 228
5. D.G. Bichet Nephrogenic diabetes insipidus Advances in Chronic Kidney Disease 13 2006 96 104
6. S.P. Bottomley The structural diversity in alpha1-antitrypsin misfolding EMBO Reports 12 2011 983 984 PMC3185355
7. P.M. Conn, and J.A. Janovick Pharmacoperone identification for therapeutic rescue of misfolded mutant proteins Frontiers in Endocrinology (Lausanne) 2 2011
8. P.M. Conn, A. Leanos-Miranda, and J.A. Janovick Protein origami: Therapeutic rescue of misfolded gene products Molecular Interventions 2 2002 308 316
9. P.M. Conn, and A. Ulloa-Aguirre Trafficking of G-protein-coupled receptors to the plasma membrane: Insights for pharmacoperone drugs Trends in Endocrinology and Metabolism 21 2010 190 197 PMC2831145
10. P.M. Conn, A. Ulloa-Aguirre, J. Ito, and J.A. Janovick G protein-coupled receptor trafficking in health and disease: Lessons learned to prepare for therapeutic mutant rescue in vivo Pharmacological Reviews 59 2007 225 250
11. R.L. Dormer, R. Derand, C.M. McNeilly, Y. Mettey, L. Bulteau-Pignoux, and T. Metaye Correction of delF508-CFTR activity with benzo(c)quinolizinium compounds through facilitation of its processing in cystic fibrosis airway cells Journal of Cell Science 114 2001 4073 4081
12. J.D. Emerson, and J. Strenio Boxplots and batch comparison D.C. Hoaglin, F. Mosteller, J. Tukey, Understanding robust and exploratory data analysis 1983 John Wiley & Sons New York 59 96
13. J.Q. Fan A contradictory treatment for lysosomal storage disorders: Inhibitors enhance mutant enzyme activity Trends in Pharmacological Sciences 24 2003 355 360
14. M. Feldhammer, S. Durand, and A.V. Pshezhetsky Protein misfolding as an underlying molecular defect in mucopolysaccharidosis III type C PloS One 4 2009 e7434
15. G. Forloni, L. Terreni, I. Bertani, S. Fogliarino, R. Invernizzi, and A. Assini Protein misfolding in Alzheimer's and Parkinson's disease: Genetics and molecular mechanisms Neurobiology of Aging 23 2002 957 976
16. L.J. Galietta, M.F. Springsteel, M. Eda, E.J. Niedzinski, K. By, and M.J. Haddadin Novel CFTR chloride channel activators identified by screening of combinatorial libraries based on flavone and benzoquinolizinium lead compounds The Journal of Biological Chemistry 276 2001 19723 19728
17. C.W. Gruber, M. Muttenthaler, and M. Freissmuth Ligand-based peptide design and combinatorial peptide libraries to target G protein-coupled receptors Current Pharmaceutical Design 16 2010 3071 3088
18. V. Heiser, E. Scherzinger, A. Boeddrich, E. Nordhoff, R. Lurz, and N. Schugardt Inhibition of huntingtin fibrillogenesis by specific antibodies and small molecules: Implications for Huntington's disease therapy Proceedings of the National Academy of Sciences of the United States of America 97 2000 6739 6744
19. J.A. Janovick, S.P. Brothers, A. Cornea, E. Bush, M.T. Goulet, and W.T. Ashton Refolding of misfolded mutant GPCR: Post-translational pharmacoperone action in vitro Molecular and Cellular Endocrinology 272 2007 77 85 PMC2169380
20. J.A. Janovick, M. Goulet, E. Bush, J. Greer, D.G. Wettlaufer, and P.M. Conn Structure-activity relations of successful pharmacologic chaperones for rescue of naturally occurring and manufactured mutants of the gonadotropin-releasing hormone receptor The Journal of Pharmacology and Experimental Therapeutics 305 2003 608 614
21. J.A. Janovick, B.S. Park, and P.M. Conn Therapeutic rescue of misfolded mutants: Validation of primary high throughput screens for identification of pharmacoperone drugs PloS One 6 2011 e22784 PMC3144936
22. M. Kumar, K. Hsiao, J. Vidugiriene, and S.A. Goueli A bioluminescent-based, HTS-compatible assay to monitor G-protein-coupled receptor modulation of cellular cyclic AMP Assay and Drug Development Technologies 5 2007 237 245
23. F. Madoux, S. Simanski, P. Chase, J.K. Mishra, W.R. Roush, and N.G. Ayad An ultra-high throughput cell-based screen for wee1 degradation inhibitors Journal of Biomolecular Screening 15 2010 907 917 3082437
24. S.J. Marciniak, and D. Ron Endoplasmic reticulum stress signaling in disease Physiological Reviews 86 2006 1133 1149
25. E. Martikkala, A. Rozwandowicz-Jansen, P. Hanninen, U. Petaja-Repo, and H. Harma A homogeneous single-label time-resolved fluorescence cAMP assay Journal of Biomolecular Screening 16 2011 356 362
26. J.P. Morello, and D.G. Bichet Nephrogenic diabetes insipidus Annual Review of Physiology 63 2001 607 630
27. J.P. Morello, A. Salahpour, A. Laperriere, V. Bernier, M.F. Arthus, and M. Lonergan Pharmacological chaperones rescue cell-surface expression and function of misfolded V2 vasopressin receptor mutants The Journal of Clinical Investigation 105 2000 887 895
28. P.J. Muchowski, and J.L. Wacker Modulation of neurodegeneration by molecular chaperones Nature Reviews. Neuroscience 6 2005 11 22
29. S.M. Noorwez, R. Malhotra, J.H. McDowell, K.A. Smith, M.P. Krebs, and S. Kaushal Retinoids assist the cellular folding of the autosomal dominant retinitis pigmentosa opsin mutant P23H The Journal of Biological Chemistry 279 2004 16278 16284
30. J. Pantel, S.Y. Williams, D. Mi, J. Sebag, J.D. Corbin, and C.D. Weaver Development of a high throughput screen for allosteric modulators of melanocortin-4 receptor signaling using a real time cAMP assay European Journal of Pharmacology 660 2011 139 147 3175485
31. Y. Peng, C. Li, L. Chen, S. Sebti, and J. Chen Rescue of mutant p53 transcription function by ellipticine Oncogene 22 2003 4478 4487
32. B. Permanne, C. Adessi, G.P. Saborio, S. Fraga, M.J. Frossard, and J. Van Dorpe Reduction of amyloid load and cerebral damage in a transgenic mouse model of Alzheimer's disease by treatment with a beta-sheet breaker peptide The FASEB Journal 16 2002 860 862
33. L. Prystay, A. Gagne, P. Kasila, L.A. Yeh, and P. Banks Homogeneous cell-based fluorescence polarization assay for the direct detection of cAMP Journal of Biomolecular Screening 6 2001 75 82
34. S.A. Ranadive, B. Ersoy, H. Favre, C.C. Cheung, S.M. Rosenthal, and W.L. Miller Identification, characterization and rescue of a novel vasopressin-2 receptor mutation causing nephrogenic diabetes insipidus Clinical Endocrinology 71 2009 388 393
35. D. Ron Translational control in the endoplasmic reticulum stress response The Journal of Clinical Investigation 110 2002 1383 1388
36. D. Ron, and S.R. Hubbard How IRE1 reacts to ER stress Cell 132 2008 24 26
37. C. Serradeil-Le Gal An overview of SR121463, a selective non-peptide vasopressin V(2) receptor antagonist Cardiovascular Drug Reviews 19 2001 201 214
38. C. Soto, R.J. Kascsak, G.P. Saborio, P. Aucouturier, T. Wisniewski, and F. Prelli Reversion of prion protein conformational changes by synthetic beta-sheet breaker peptides Lancet 355 2000 192 197
39. A. Ulloa-Aguirre, J.A. Janovick, A. Leanos-Miranda, and P.M. Conn Misrouted cell surface receptors as a novel disease aetiology and potential therapeutic target: The case of hypogonadotropic hypogonadism due to gonadotropin-releasing hormone resistance Expert Opinion on Therapeutic Targets 7 2003 175 185
40. J.H. Zhang, T.D. Chung, and K.R. Oldenburg A simple statistical parameter for use in evaluation and validation of high throughput screening assays Journal of Biomolecular Screening 4 1999 67 73
41. J.Y. Zhang, D.M. Kowal, S.P. Nawoschik, J. Dunlop, M.H. Pausch, and R. Peri Development of an improved IP(1) assay for the characterization of 5-HT(2C) receptor ligands Assay and Drug Development Technologies 8 2010 106 113
42. X.M. Zhang, X.T. Wang, H. Yue, S.W. Leung, P.H. Thibodeau, and P.J. Thomas Organic solutes rescue the functional defect in delta F508 cystic fibrosis transmembrane conductance regulator The Journal of Biological Chemistry 278 2003 51232 51242