{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,30]],"date-time":"2026-06-30T15:24:28Z","timestamp":1782833068802,"version":"3.54.5"},"publisher-location":"Cham","reference-count":83,"publisher":"Springer International Publishing","isbn-type":[{"value":"9783319741635","type":"print"},{"value":"9783319741642","type":"electronic"}],"license":[{"start":{"date-parts":[[2017,1,1]],"date-time":"2017-01-01T00:00:00Z","timestamp":1483228800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2017]]},"DOI":"10.1007\/164_2017_50","type":"book-chapter","created":{"date-parts":[[2017,9,22]],"date-time":"2017-09-22T08:50:37Z","timestamp":1506070237000},"page":"63-83","source":"Crossref","is-referenced-by-count":12,"title":["Pharmacological Chaperones as Potential Therapeutic Strategies for Misfolded Mutant Vasopressin Receptors"],"prefix":"10.1007","author":[{"given":"Bernard","family":"Mouillac","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christiane","family":"Mendre","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","reference":[{"issue":"10","key":"50_CR1","doi-asserted-by":"crossref","first-page":"1861","DOI":"10.1681\/ASN.V9101861","volume":"9","author":"Y Ala","year":"1998","unstructured":"Ala Y, Morin D, Mouillac B et al (1998) Functional studies of twelve mutant V2 vasopressin receptors related to nephrogenic diabetes insipidus: molecular basis of a mild clinical phenotype. J Am Soc Nephrol 9(10):1861\u20131872","journal-title":"J Am Soc Nephrol"},{"issue":"3","key":"50_CR2","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1093\/eurjhf\/hfq226","volume":"13","author":"D Aronson","year":"2011","unstructured":"Aronson D, Verbalis JG, Mueller M et al (2011) Short- and long-term treatment of dilutional hyponatraemia with satavaptan, a selective arginine-vasopressin V2 receptor antagonist: the DILIPO study. Eur J Heart Fail 13(3):327\u2013336","journal-title":"Eur J Heart Fail"},{"issue":"51","key":"50_CR3","doi-asserted-by":"publisher","first-page":"42198","DOI":"10.1074\/jbc.M510180200","volume":"280","author":"RJ Auzan","year":"2005","unstructured":"Auzan RJ, Ventura MA, Clauser E (2005) Mechanisms of cell-surface rerouting of an endoplasmic reticulum-retained mutant of the vasopressin V1b\/V3 receptor by a pharmacological chaperone. J Biol Chem 280(51):42198\u201342206","journal-title":"J Biol Chem"},{"issue":"5","key":"50_CR4","doi-asserted-by":"publisher","first-page":"528","DOI":"10.1016\/j.coph.2004.08.001","volume":"4","author":"V Bernier","year":"2004","unstructured":"Bernier V, Bichet DG, Bouvier M (2004a) Pharmacological chaperone action on G protein-coupled receptors. Curr Opin Pharmacol 4(5):528\u2013533","journal-title":"Curr Opin Pharmacol"},{"issue":"8","key":"50_CR5","doi-asserted-by":"publisher","first-page":"2074","DOI":"10.1210\/me.2004-0080","volume":"18","author":"V Bernier","year":"2004","unstructured":"Bernier V, Lagac\u00e9 M, Lonergan M et al (2004b) Functional rescue of the constitutively internalized V2 vasopressin receptor mutant R137H by the pharmacological chaperone action of SR49059. Mol Endocrinol 18(8):2074\u20132084","journal-title":"Mol Endocrinol"},{"issue":"1","key":"50_CR6","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1681\/ASN.2005080854","volume":"17","author":"V Bernier","year":"2006","unstructured":"Bernier V, Morello JP, Zarruk A et al (2006) Pharmacologic chaperones as a potential treatment for X-linked nephrogenic diabetes insipidus. J Am Soc Nephrol 17(1):233\u2013243","journal-title":"J Am Soc Nephrol"},{"issue":"2","key":"50_CR7","first-page":"278","volume":"55","author":"DG Bichet","year":"1994","unstructured":"Bichet DG, Birnbaumer M, Lonergan M et al (1994) Nature and recurrence of AVPR2 mutations in X-linked nephrogenic diabetes insipidus. Am J Hum Genet 55(2):278\u2013286","journal-title":"Am J Hum Genet"},{"issue":"6376","key":"50_CR8","doi-asserted-by":"publisher","first-page":"333","DOI":"10.1038\/357333a0","volume":"357","author":"M Birnbaumer","year":"1992","unstructured":"Birnbaumer M, Seibold A, Gilbert S et al (1992) Molecular cloning of the receptor for human antidiuretic hormone. Nature 357(6376):333\u2013335","journal-title":"Nature"},{"issue":"1","key":"50_CR9","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1159\/000245059","volume":"114","author":"D Bockenhauer","year":"2010","unstructured":"Bockenhauer D, Carpentier E, Rochdi D et al (2010) Vasopressin type 2 receptor V88M mutation: molecular basis of partial and complete nephrogenic diabetes insipidus. Nephron Physiol 114(1):1\u201310","journal-title":"Nephron Physiol"},{"issue":"8","key":"50_CR10","doi-asserted-by":"publisher","first-page":"1297","DOI":"10.1007\/s00467-013-2526-4","volume":"29","author":"D Bockenhauer","year":"2014","unstructured":"Bockenhauer D, Bichet DG (2014) Urinary concentration: different ways to open and close the tap. Pediatr Nephrol 29(8):1297\u20131303","journal-title":"Pediatr Nephrol"},{"key":"50_CR11","doi-asserted-by":"publisher","first-page":"283","DOI":"10.1146\/annurev.med.57.110104.115650","volume":"57","author":"RO Brady","year":"2006","unstructured":"Brady RO (2006) Enzyme replacement for lysosomal diseases. Annu Rev Med 57:283\u2013296","journal-title":"Annu Rev Med"},{"issue":"2","key":"50_CR12","doi-asserted-by":"publisher","first-page":"839","DOI":"10.1124\/jpet.105.088914","volume":"315","author":"AA Carter","year":"2005","unstructured":"Carter AA, Hill SJ (2005) Characterization of isoprenaline- and salmeterol-stimulated interactions between beta2-adrenoceptors and beta-arrestin 2 using beta-galactosidase complementation in C2C12 cells. J Pharmacol Exp Ther 315(2):839\u2013848","journal-title":"J Pharmacol Exp Ther"},{"issue":"7","key":"50_CR13","doi-asserted-by":"publisher","first-page":"1331","DOI":"10.1111\/j.1742-4658.2006.05181.x","volume":"273","author":"TK Chaudhuri","year":"2006","unstructured":"Chaudhuri TK, Paul S (2006) Protein-misfolding diseases and chaperone-based therapeutic approaches. FEBS J 273(7):1331\u20131349","journal-title":"FEBS J"},{"issue":"16","key":"50_CR14","doi-asserted-by":"publisher","first-page":"7141","DOI":"10.1021\/jm300603y","volume":"55","author":"Xin Chen","year":"2012","unstructured":"Chen X, Sassano MF, Zheng L et al (2012) Structure-functional selectivity relationship studies of \u03b2-arrestin-biased dopamine D2 receptor agonists. J Med Chem 55(16):7141\u20137153","journal-title":"Journal of Medicinal Chemistry"},{"issue":"6968","key":"50_CR15","doi-asserted-by":"publisher","first-page":"905","DOI":"10.1038\/nature02265","volume":"426","author":"FE Cohen","year":"2003","unstructured":"Cohen FE, Kelly LW (2003) Therapeutic approaches to protein-misfolding diseases. Nature 426(6968):905\u2013909","journal-title":"Nature"},{"issue":"3","key":"50_CR16","doi-asserted-by":"publisher","first-page":"225","DOI":"10.1124\/pr.59.3.2","volume":"59","author":"PM Conn","year":"2007","unstructured":"Conn PM, Ulloa-Aguire A, Ito J et al (2007) G protein-coupled receptor trafficking in health and disease: lessons learned to prepare for therapeutic mutant rescue in vivo. Pharmacol Rev 59(3):225\u2013250","journal-title":"Pharmacol Rev"},{"issue":"3","key":"50_CR17","doi-asserted-by":"publisher","first-page":"190","DOI":"10.1016\/j.tem.2009.11.003","volume":"21","author":"PM Conn","year":"2010","unstructured":"Conn PM, Ulloa-Aguirre A (2010) Trafficking of G protein-coupled receptors to the plasma membrane: insights from pharmacoperone drugs. Trends Endocrinol Metab 21(3):190\u2013197","journal-title":"Trends Endocrinol Metab"},{"key":"50_CR18","doi-asserted-by":"publisher","first-page":"109","DOI":"10.1016\/B978-0-12-385952-5.00008-7","volume":"62","author":"PM Conn","year":"2011","unstructured":"Conn PM, Ulloa-Aguirre A (2011) Pharmacological chaperones for misfolded gonadotropin-releasing hormone receptors. Adv Pharmacol 62:109\u2013141","journal-title":"Adv Pharmacol"},{"issue":"8","key":"50_CR19","doi-asserted-by":"publisher","first-page":"930","DOI":"10.1177\/1087057113483559","volume":"18","author":"PM Conn","year":"2013","unstructured":"Conn PM, Smith E, Hodder PS et al (2013) High throughput screen for pharmacoperones of the vasopressin type 2 receptor. J Biomol Screen 18(8):930\u2013937","journal-title":"J Biomol Screen"},{"key":"50_CR20","doi-asserted-by":"publisher","first-page":"38","DOI":"10.1016\/j.phrs.2013.12.004","volume":"83","author":"PM Conn","year":"2014","unstructured":"Conn PM, Smithson DC, Hodder PS et al (2014) Transitioning pharmacoperones to therapeutic use: in vivo proof-of-principle and design of high throughput screens. Pharmacol Res 83:38\u201351","journal-title":"Pharmacol Res"},{"issue":"8","key":"50_CR21","doi-asserted-by":"publisher","first-page":"498","DOI":"10.1016\/j.tips.2015.05.004","volume":"36","author":"PM Conn","year":"2015","unstructured":"Conn PM, Spicer TP, Scampavia L et al (2015) Assays strategies for identification of therapeutic leads that target protein trafficking. Trends Pharmacol Sci 36(8):498\u2013505","journal-title":"Trends Pharmacol Sci"},{"issue":"39","key":"50_CR22","doi-asserted-by":"publisher","first-page":"27849","DOI":"10.1074\/jbc.M112.445098","volume":"288","author":"TN Feinstein","year":"2013","unstructured":"Feinstein TN, Yui N, Webber MJ et al (2013) Noncanonical control of vasopressin receptor type 2 signaling by retromer and arrestin. J Biol Chem 288(39):27849\u201327860","journal-title":"J Biol Chem"},{"issue":"1","key":"50_CR23","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0031-6997(24)01478-9","volume":"53","author":"SS Ferguson","year":"2001","unstructured":"Ferguson SS (2001) Evolving concepts in G protein-coupled receptor endocytosis: the role in receptor desensitization and signaling. Pharmacol Rev 53(1):1\u201324","journal-title":"Pharmacol Rev"},{"issue":"8","key":"50_CR24","doi-asserted-by":"publisher","first-page":"423","DOI":"10.1016\/j.tips.2007.06.005","volume":"28","author":"S Galandrin","year":"2007","unstructured":"Galandrin S, Oligny-Longpr\u00e9 G, Bouvier M (2007) The evasine nature of drug efficacy: implications for drug discovery. Trends Pharmacol Sci 28(8):423\u2013430","journal-title":"Trends Pharmacol Sci"},{"key":"50_CR25","doi-asserted-by":"publisher","first-page":"91","DOI":"10.1186\/1750-1172-7-91","volume":"7","author":"DP Germain","year":"2012","unstructured":"Germain DP, Giugliani R, Hughes DA et al (2012) Safety and pharmacodynamic effects of a pharmacological chaperone on \u03b1-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies. Orphanet J Rare Dis 7:91","journal-title":"Orphanet J Rare Dis"},{"issue":"6","key":"50_CR26","doi-asserted-by":"publisher","first-page":"545","DOI":"10.1056\/NEJMoa1510198","volume":"375","author":"DP Germain","year":"2016","unstructured":"Germain DP, Hughes DA, Nicholls K et al (2016) Treatment of Fabry\u2019s disease with the pharmacologic chaperone migalastat. N Engl J Med 375(6):545\u2013555","journal-title":"N Engl J Med"},{"issue":"2","key":"50_CR27","doi-asserted-by":"publisher","first-page":"549","DOI":"10.1124\/mol.106.028258","volume":"71","author":"CE Groer","year":"2007","unstructured":"Groer CE, Tidgewell K, Moyer RA et al (2007) An opioid agonist that does not induce mu-opioid receptor-arrestin interactions or receptor internalization. Mol Pharmacol 71(2):549\u2013557","journal-title":"Mol Pharmacol"},{"issue":"21","key":"50_CR28","doi-asserted-by":"publisher","first-page":"14604","DOI":"10.1074\/jbc.M511610200","volume":"281","author":"SR Hawtin","year":"2006","unstructured":"Hawtin SR (2006) Pharmacological chaperone activity of SR49059 to functionally recover misfolded mutations of the vasopressin V1a receptor. J Biol Chem 281(21):14604\u201314614","journal-title":"J Biol Chem"},{"issue":"4","key":"50_CR29","doi-asserted-by":"publisher","first-page":"768","DOI":"10.1124\/mol.61.4.768","volume":"61","author":"AC Holloway","year":"2002","unstructured":"Holloway AC, Qian H, Pipolo L et al (2002) Side-chain substitutions within angiotensin II reveal different requirements for signaling, internalization, and phosphorylation of type 1A angiotensin receptors. Mol Pharmacol 61(4):768\u2013777","journal-title":"Mol Pharmacol"},{"issue":"7","key":"50_CR30","doi-asserted-by":"publisher","first-page":"3255","DOI":"10.1210\/jcem.87.7.8582","volume":"87","author":"JA Janovick","year":"2002","unstructured":"Janovick JA, Maya-Nunez G, Conn PM (2002) Rescue of hypogonadotropic hypogonadism-causing and manufactured GnRH receptor mutants by a specific protein-folding template: misrouted proteins as a novel disease etiology and therapeutic target. J Clin Endocrinol Metab 87(7):3255\u20133262","journal-title":"J Clin Endocrinol Metab"},{"issue":"1\u20132","key":"50_CR31","doi-asserted-by":"publisher","first-page":"84","DOI":"10.1016\/j.mce.2008.09.015","volume":"298","author":"JA Janovick","year":"2009","unstructured":"Janovick JA, Maya-Nunez G, Ulloa-Aguire A et al (2009) Increased plasma membrane expression of human follicle-stimulating hormone receptor by a small molecule thienopyr(im)idine. Mol Cell Endocrinol 298(1\u20132):84\u201388","journal-title":"Mol Cell Endocrinol"},{"issue":"7","key":"50_CR32","doi-asserted-by":"publisher","first-page":"e22784","DOI":"10.1371\/journal.pone.0022784","volume":"6","author":"JA Janovick","year":"2011","unstructured":"Janovick JA, Park BS, Conn PM (2011) Therapeutic rescue of misfolded mutants: validation of primary high throughput screens for identification of pharmacoperone drugs. PLoS One 6(7):e22784","journal-title":"PLoS One"},{"issue":"52","key":"50_CR33","doi-asserted-by":"publisher","first-page":"21030","DOI":"10.1073\/pnas.1315194110","volume":"110","author":"JA Janovick","year":"2013","unstructured":"Janovick JA, Stewart MD, Jacob D et al (2013) Restoration of testis function in hypogonadotropic hypogonadal mice harboring a misfolded GnRHR mutant by pharmacoperone drug therapy. Proc Natl Acad Sci U S A 110(52):21030\u201321035","journal-title":"Proc Natl Acad Sci U S A"},{"key":"50_CR34","doi-asserted-by":"publisher","first-page":"176","DOI":"10.1016\/j.mce.2016.07.003","volume":"434","author":"JA Janovick","year":"2016","unstructured":"Janovick JA, Spicer TP, Smith E et al (2016) Receptor antagonism\/agonism can be uncoupled from pharmacoperone activity. Mol Cell Endocrinol 434:176\u2013185","journal-title":"Mol Cell Endocrinol"},{"issue":"8","key":"50_CR35","doi-asserted-by":"publisher","first-page":"e0181830","DOI":"10.1371\/journal.pone.0181830","volume":"12","author":"JA Janovick","year":"2017","unstructured":"Janovick JA, Spicer TP, Bannister TD et al (2017) Pharmacoperone rescue of vasopressin 2 receptor mutants reveals unexpected constitutive activity and coupling bias. PLoS One 12(8):e0181830","journal-title":"PLoS One"},{"issue":"10","key":"50_CR36","doi-asserted-by":"publisher","first-page":"2190","DOI":"10.1681\/ASN.2008121289","volume":"20","author":"F Jean-Alphonse","year":"2009","unstructured":"Jean-Alphonse F, Perkovska S, Frantz MC et al (2009) Biased agonist pharmacochaperones of the AVP V2 receptor may treat congenital nephrogenic diabetes insipidus. J Am Soc Nephrol 20(10):2190\u20132203","journal-title":"J Am Soc Nephrol"},{"issue":"1","key":"50_CR37","doi-asserted-by":"publisher","first-page":"85","DOI":"10.1006\/excr.1997.3581","volume":"234","author":"LE Karageorgos","year":"1997","unstructured":"Karageorgos LE, Isaac EL, Brooks DA et al (1997) Lysosomal biogenesis in lysosomal storage disorders. Exp Cell Res 234(1):85\u201397","journal-title":"Exp Cell Res"},{"issue":"1","key":"50_CR38","doi-asserted-by":"publisher","first-page":"116","DOI":"10.1073\/pnas.93.1.116","volume":"93","author":"KL Laugwitz","year":"1996","unstructured":"Laugwitz KL, Allgeier A, Offermanns S et al (1996) The human thyrotropin receptor: a heptahelical receptor capable of stimulating members of all four G protein families. Proc Natl Acad Sci U S A 93(1):116\u2013120","journal-title":"Proc Natl Acad Sci U S A"},{"key":"50_CR39","doi-asserted-by":"publisher","first-page":"10","DOI":"10.1016\/j.phrs.2014.01.005","volume":"83","author":"NJ Leidenheimer","year":"2014","unstructured":"Leidenheimer NJ, Ryder KG (2014) Pharmacological chaperoning: a primer on mechanism and pharmacology. Pharmacol Res 83:10\u201319","journal-title":"Pharmacol Res"},{"issue":"6376","key":"50_CR40","doi-asserted-by":"publisher","first-page":"526","DOI":"10.1038\/357336a0","volume":"357","author":"SJ Lolait","year":"1992","unstructured":"Lolait SJ, Carroll AM, McBride OW et al (1992) Cloning and characterization of a vasopressin V2 receptor and possible link to nephrogenic diabetes insipidus. Nature 357(6376):526\u2013529","journal-title":"Nature"},{"issue":"2","key":"50_CR41","doi-asserted-by":"publisher","first-page":"709","DOI":"10.1074\/jbc.272.2.709","volume":"272","author":"TW Loo","year":"1997","unstructured":"Loo TW, Clarke DM (1997) Correction of defective protein kinesis of human P-glycoprotein mutants by substrates and modulators. J Biol Chem 272(2):709\u2013712","journal-title":"J Biol Chem"},{"issue":"5","key":"50_CR42","doi-asserted-by":"publisher","first-page":"393","DOI":"10.1111\/j.1365-2826.2010.01983.x","volume":"22","author":"EL Los","year":"2010","unstructured":"Los EL, Deen PMT, Robben JH (2010) Potential of nonpeptide (ant)agonists to rescue vasopressin V2 receptor mutants for the treatment of X-linked nephrogenic diabetes insipidus. J Neuroendocrinol 22(5):393\u2013399","journal-title":"J Neuroendocrinol"},{"issue":"2","key":"50_CR43","doi-asserted-by":"publisher","first-page":"305","DOI":"10.1124\/pr.109.002436","volume":"62","author":"LM Luttrell","year":"2010","unstructured":"Luttrell LM, Gesty-Palmer D (2010) Beyond desensitization: physiological relevance of arrestin-dependent signaling. Pharmacol Rev 62(2):305\u2013330","journal-title":"Pharmacol Rev"},{"issue":"3","key":"50_CR44","doi-asserted-by":"publisher","first-page":"281","DOI":"10.1210\/me.2013-1314","volume":"28","author":"LM Lutrell","year":"2014","unstructured":"Lutrell LM (2014) More than just a hammer: ligand \u201cbias\u201d and pharmaceutical discovery. Mol Endocrinol 28(3):281\u2013294","journal-title":"Mol Endocrinol"},{"issue":"3","key":"50_CR45","doi-asserted-by":"publisher","first-page":"579","DOI":"10.1124\/mol.115.099630","volume":"88","author":"LM Luttrell","year":"2015","unstructured":"Luttrell LM, Maudsley S, Bohn LM (2015) Fulfilling the promise of \u201cbiased\u201d G protein-coupled receptor agonism. Mol Pharmacol 88(3):579\u2013588","journal-title":"Mol Pharmacol"},{"issue":"6\u20137","key":"50_CR46","doi-asserted-by":"publisher","first-page":"627","DOI":"10.1051\/medsci\/2010266-7627","volume":"26","author":"C Mendre","year":"2010","unstructured":"Mendre C, Mouillac B (2010) Pharmacological chaperones: a potential therapeutic treatment for conformational diseases. Med Sci (Paris) 26(6\u20137):627\u2013635","journal-title":"Med Sci (Paris)"},{"issue":"2","key":"50_CR47","doi-asserted-by":"publisher","first-page":"278","DOI":"10.1210\/er.2012-1044","volume":"34","author":"HB Moeller","year":"2013","unstructured":"Moeller HB, Rittig S, Fenton RA (2013) Nephrogenic diabetes insipidus: essential insights into the molecular background and potential therapies for treatment. Endocr Rev 34(2):278\u2013301","journal-title":"Endocr Rev"},{"issue":"7","key":"50_CR48","doi-asserted-by":"publisher","first-page":"887","DOI":"10.1172\/JCI8688","volume":"105","author":"JP Morello","year":"2000","unstructured":"Morello JP, Salahpour A, Laperri\u00e8re A et al (2000a) Pharmacological chaperones rescue cell-surface expression and function of misfolded V2 vasopressin receptor mutants. J Clin Invest 105(7):887\u2013895","journal-title":"J Clin Invest"},{"issue":"12","key":"50_CR49","doi-asserted-by":"publisher","first-page":"466","DOI":"10.1016\/S0165-6147(00)01575-3","volume":"21","author":"JP Morello","year":"2000","unstructured":"Morello JP, Pet\u00e4j\u00e4-Repo UE, Bichet DG et al (2000b) Pharmacological chaperones: a new twist on receptor folding. Trends Pharmacol Sci 21(12):466\u2013469","journal-title":"Trends Pharmacol Sci"},{"key":"50_CR50","doi-asserted-by":"publisher","first-page":"607","DOI":"10.1146\/annurev.physiol.63.1.607","volume":"63","author":"JP Morello","year":"2001","unstructured":"Morello JP, Bichet DG (2001) Nephrogenic diabetes insipidus. Annu Rev Physiol 63:607\u2013630","journal-title":"Annu Rev Physiol"},{"key":"50_CR51","doi-asserted-by":"publisher","first-page":"74","DOI":"10.1016\/j.phrs.2013.10.007","volume":"83","author":"B Mouillac","year":"2014","unstructured":"Mouillac B, Mendre C (2014) Vasopressin receptors and pharmacological chaperones: from functional rescue to promising therapeutic strategies. Pharmacol Res 83:74\u201378","journal-title":"Pharmacol Res"},{"issue":"17","key":"50_CR52","doi-asserted-by":"publisher","first-page":"7172","DOI":"10.1073\/pnas.1015723108","volume":"108","author":"CL Newton","year":"2011","unstructured":"Newton CL, Whay AM, McArdle CA et al (2011) Rescue of expression and signaling of human luteinizing hormone G protein-coupled receptor mutants with an allosterically binding small-molecule agonist. Proc Natl Acad Sci U S A 108(17):7172\u20137176","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"5","key":"50_CR53","doi-asserted-by":"crossref","first-page":"890","DOI":"10.1016\/S0026-895X(25)10203-4","volume":"45","author":"S Offermanns","year":"1994","unstructured":"Offermanns S, Wieland T, Homann D et al (1994) Transfected muscarinic acetylcholine receptors selectively couple to Gi-type G proteins and Gq\/11. Mol Pharmacol 45(5):890\u2013898","journal-title":"Mol Pharmacol"},{"issue":"7","key":"50_CR54","doi-asserted-by":"publisher","first-page":"1138","DOI":"10.1038\/mt.2015.62","volume":"23","author":"G Parenti","year":"2015","unstructured":"Parenti G, Andria G, Valenzano KJ (2015) Pharmacological chaperone therapy: preclinical development, clinical translation, and prospects for the treatment of lysosomal storage disorders. Mol Ther 23(7):1138\u20131148","journal-title":"Mol Ther"},{"issue":"2","key":"50_CR55","first-page":"102","volume":"25","author":"EJ Parkinson-Lawrence","year":"2010","unstructured":"Parkinson-Lawrence EJ, Shandala T, Prodoehl M et al (2010) Lysosomal storage disease: revealing lysosomal function and physiology. Physiology (Bethesda) 25(2):102\u2013115","journal-title":"Physiology (Bethesda)"},{"issue":"457","key":"50_CR56","doi-asserted-by":"publisher","first-page":"88","DOI":"10.1111\/j.1651-2227.2008.00656.x","volume":"97","author":"FM Platt","year":"2008","unstructured":"Platt FM, Jeyakumar M (2008) Substrate reduction therapy. Acta Paediatr 97(457):88\u201393","journal-title":"Acta Paediatr"},{"issue":"17","key":"50_CR57","doi-asserted-by":"publisher","first-page":"6733","DOI":"10.1073\/pnas.1201093109","volume":"109","author":"R Rahmeh","year":"2012","unstructured":"Rahmeh R, Damian M, Cottet M et al (2012) Structural insights into biased G protein-coupled receptor signaling revealed by fluorescence spectroscopy. Proc Natl Acad Sci U S A 109(17):6733\u20136738","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"5","key":"50_CR58","doi-asserted-by":"publisher","first-page":"373","DOI":"10.1038\/nrd3024","volume":"9","author":"S Rajagopal","year":"2010","unstructured":"Rajagopal S, Rajagopal K, Lefkowitz RJ (2010) Teaching old receptors new tricks: biasing seven-transmembrane receptors. Nat Rev Drug Discov 9(5):373\u2013386","journal-title":"Nat Rev Drug Discov"},{"issue":"1","key":"50_CR59","doi-asserted-by":"publisher","first-page":"179","DOI":"10.1146\/annurev.pharmtox.010909.105800","volume":"52","author":"Eric Reiter","year":"2012","unstructured":"Reiter E, Ahn S, Shukla AK et al (2012) Molecular mechanism of \u03b2-arrestin-biased agonism at seven-transmembrane receptors. Annu Rev Pharmacol Toxicol 52:179\u2013197","journal-title":"Annual Review of Pharmacology and Toxicology"},{"issue":"1","key":"50_CR60","doi-asserted-by":"publisher","first-page":"379","DOI":"10.1091\/mbc.E05-06-0579","volume":"17","author":"JH Robben","year":"2006","unstructured":"Robben JH, Sze M, Knoers NV et al (2006) Rescue of vasopressin V2 receptor mutants by chemical chaperones: specificity and mechanism. Mol Biol Cell 17(1):379\u2013386","journal-title":"Mol Biol Cell"},{"issue":"1","key":"50_CR61","doi-asserted-by":"publisher","first-page":"F253","DOI":"10.1152\/ajprenal.00247.2006","volume":"292","author":"JH Robben","year":"2007","unstructured":"Robben JH, Sze M, Knoers NV et al (2007) Functional rescue of vasopressin V2 receptor mutants in MDCK cells by pharmacochaperones: relevance to therapy of nephrogenic diabetes insipidus. Am J Physiol Renal Physiol 292(1):F253\u2013F260","journal-title":"Am J Physiol Renal Physiol"},{"issue":"29","key":"50_CR62","doi-asserted-by":"publisher","first-page":"12195","DOI":"10.1073\/pnas.0900130106","volume":"106","author":"JH Robben","year":"2009","unstructured":"Robben JH, Kortenoeven MLA, Sze M et al (2009) Intracellular activation of vasopressin V2 receptor mutants in nephrogenic diabetes insipidus by nonpeptide agonists. Proc Natl Acad Sci U S A 106(29):12195\u201312200","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"6392","key":"50_CR63","doi-asserted-by":"publisher","first-page":"233","DOI":"10.1038\/359233a0","volume":"359","author":"W Rosenthal","year":"1992","unstructured":"Rosenthal W, Seibold A, Antaramian A et al (1992) Molecular identification of the gene responsible for congenital nephrogenic diabetes insipidus. Nature 359(6392):233\u2013235","journal-title":"Nature"},{"issue":"3","key":"50_CR64","doi-asserted-by":"crossref","first-page":"1039","DOI":"10.1016\/S0022-3565(25)12378-1","volume":"276","author":"S Sagan","year":"1996","unstructured":"Sagan S, Chassaing G, Pradier L et al (1996) Tachykinin peptides affect differently the second messenger pathways after binding to CHO-expressed human NK-1 receptors. J Pharmacol Exp Ther 276(3):1039\u20131048","journal-title":"J Pharmacol Exp Ther"},{"issue":"2","key":"50_CR65","doi-asserted-by":"publisher","first-page":"635","DOI":"10.1074\/jbc.271.2.635","volume":"271","author":"S Sato","year":"1996","unstructured":"Sato S, Ward CL, Krouse ME et al (1996) Glycerol reverses the misfolding phenotype of the most common cystic fibrosis mutation. J Biol Chem 271(2):635\u2013638","journal-title":"J Biol Chem"},{"issue":"20","key":"50_CR66","doi-asserted-by":"publisher","first-page":"2099","DOI":"10.1056\/NEJMoa065181","volume":"355","author":"RW Schrier","year":"2006","unstructured":"Schrier RW, Gross P, Gheorghiade M (2006) Tolvaptan, a selective oral vasopressin V2-receptor antagonist, for hyponatremia. New Engl J Med 355(20):2099\u20132112","journal-title":"New Engl J Med"},{"issue":"16","key":"50_CR67","doi-asserted-by":"publisher","first-page":"5101","DOI":"10.1021\/jm800258p","volume":"51","author":"G Semple","year":"2008","unstructured":"Semple G, Skinner PJ, Gharbaoui T et al (2008) 3-(1H-tetrazol-5-yl)-1,4,5,6-tetrahydro-cyclopentapyrazole (MK-0354): a partial agonist of the nicotinic acid receptor, G-protein coupled receptor 109a, with antilipolytic but no vasodilatory activity in mice. J Med Chem 51(16):5101\u20135108","journal-title":"J Med Chem"},{"issue":"12","key":"50_CR68","doi-asserted-by":"publisher","first-page":"2729","DOI":"10.1172\/JCI119098","volume":"98","author":"C Serradeil-Le Gal","year":"1996","unstructured":"Serradeil-Le Gal C, Lacour C, Valette G et al (1996) Characterization of SR 121463A, a highly potent and selective, orally active vasopressin V2 receptor antagonist. J Clin Invest 98(12):2729\u20132738","journal-title":"J Clin Invest"},{"key":"50_CR69","doi-asserted-by":"crossref","unstructured":"Shenoy S, Lefkowitz RJ (2011) \u03b2-Arrestin-mediated receptor trafficking and signal transduction. Trends Pharmacol Sci 32(9): 521-533","DOI":"10.1016\/j.tips.2011.05.002"},{"issue":"8","key":"50_CR70","doi-asserted-by":"publisher","first-page":"824","DOI":"10.1177\/1087057116653925","volume":"21","author":"E Smith","year":"2016","unstructured":"Smith E, Janovick JA, Bannister TD et al (2016) Identification of potential pharmacoperones capable of rescuing the functionality of misfolded vasopressin 2 receptor involved in nephrogenic diabetes insipidus. J Biomol Screen 21(8):824\u2013831","journal-title":"J Biomol Screen"},{"key":"50_CR71","doi-asserted-by":"publisher","first-page":"3","DOI":"10.1016\/B978-0-12-391862-8.00001-6","volume":"521","author":"DC Smithson","year":"2013","unstructured":"Smithson DC, Janovick JA, Conn PM (2013) Therapeutic rescue of misfolded\/mistrafficked mutants: automation-friendly high-throughput assays for identification of pharmacoperone drugs of GPCRs. Methods Enzymol 521:3\u201316","journal-title":"Methods Enzymol"},{"issue":"41","key":"50_CR72","doi-asserted-by":"publisher","first-page":"13453","DOI":"10.1021\/bi990437n","volume":"38","author":"H Takasu","year":"1999","unstructured":"Takasu H, Gardella TJ, Luck MD et al (1999) Amino-terminal modifications of human parathyroid hormone (PTH) selectively alter phospholipase C signaling via the type 1 PTH receptor: implications for design of signal-specific PTH ligands. Biochemistry 38(41):13453\u201313460","journal-title":"Biochemistry"},{"issue":"10","key":"50_CR73","doi-asserted-by":"publisher","first-page":"2257","DOI":"10.1172\/JCI2303","volume":"101","author":"BK Tamarappoo","year":"1998","unstructured":"Tamarappoo BK, Verkman AS (1998) Defective aquaporin-2 trafficking in nephrogenic diabetes insipidus and correction by chemical chaperones. J Clin Invest 101(10):2257\u20132267","journal-title":"J Clin Invest"},{"issue":"37","key":"50_CR74","doi-asserted-by":"publisher","first-page":"35678","DOI":"10.1074\/jbc.M301888200","volume":"278","author":"CM Tan","year":"2003","unstructured":"Tan CM, Nickols HH, Limbird LE (2003) Appropriate polarization following pharmacological rescue of V2 vasopressin receptors encoded by X-linked nephrogenic diabetes insipidus alleles involves a conformation of the receptor that also attains mature glycosylation. J Biol Chem 278(37):35678\u201335686","journal-title":"J Biol Chem"},{"issue":"1","key":"50_CR75","doi-asserted-by":"publisher","first-page":"404","DOI":"10.1124\/jpet.103.061754","volume":"309","author":"RL Thurmond","year":"2004","unstructured":"Thurmond RL, Desai PJ, Dunford PJ et al (2004) A potent and selective histamine H4 receptor antagonist with anti-inflammatory properties. J Pharmacol Exp Ther 309(1):404\u2013413","journal-title":"J Pharmacol Exp Ther"},{"issue":"3","key":"50_CR76","doi-asserted-by":"publisher","first-page":"599","DOI":"10.1097\/00000542-200609000-00026","volume":"105","author":"TA Treschan","year":"2006","unstructured":"Treschan TA, Peters J (2006) The vasopressin system. Anesthesiology 105(3):599\u2013612","journal-title":"Anesthesiology"},{"issue":"4","key":"50_CR77","doi-asserted-by":"publisher","first-page":"2043","DOI":"10.1172\/JCI118252","volume":"96","author":"H Tsukagushi","year":"1995","unstructured":"Tsukagushi H, Matsubara H, Taketani S et al (1995) Binding, intracellular transport and biosynthesis-defective mutants of vasopressin type 2 receptor in patients with X-linked nephrogenic diabetes insipidus. J Clin Invest 96(4):2043\u20132050","journal-title":"J Clin Invest"},{"key":"50_CR78","doi-asserted-by":"crossref","unstructured":"Violin JD, DeWire SM, Yamashita D et al (2010) Selectively engaging \u03b2-arrestins at the angiotensin II type 1 receptor reduces blood pressure and increases cardiac performance. J Pharmacol Exp Ther 335(3):572\u2013579","DOI":"10.1124\/jpet.110.173005"},{"issue":"12","key":"50_CR79","doi-asserted-by":"publisher","first-page":"2183","DOI":"10.1007\/s00467-012-2118-8","volume":"27","author":"D Wesche","year":"2012","unstructured":"Wesche D, Deen PMT, Knoers NV (2012) Congenital nephrogenic diabetes insipidus: the current state of affairs. Pediatr Nephrol 27(12):2183\u20132204","journal-title":"Pediatr Nephrol"},{"issue":"7","key":"50_CR80","doi-asserted-by":"publisher","first-page":"1115","DOI":"10.1210\/me.2009-0041","volume":"23","author":"E White","year":"2009","unstructured":"White E, McKenna J, Cavanaugh A et al (2009) Pharmacochaperone-mediated rescue of calcium-sensing receptor loss-of-function mutants. Mol Endocrinol 23(7):1115\u20131123","journal-title":"Mol Endocrinol"},{"issue":"42","key":"50_CR81","doi-asserted-by":"publisher","first-page":"16657","DOI":"10.1073\/pnas.0707936104","volume":"104","author":"JW Wisler","year":"2007","unstructured":"Wisler JW, DeWire SM, Whalen EJ et al (2007) A unique mechanism of beta-blocker action: carvedilol stimulates beta-arrestin signaling. Proc Natl Acad Sci U S A 104(42):16657\u201316662","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"45","key":"50_CR82","doi-asserted-by":"publisher","first-page":"47254","DOI":"10.1074\/jbc.M408154200","volume":"279","author":"S W\u00fcller","year":"2004","unstructured":"W\u00fcller S, Wiesner B, Loffler A et al (2004) Pharmacochaperones post-translationally enhance cell surface expression by increasing conformational stability of wild-type and mutant vasopressin V2 receptors. J Biol Chem 279(45):47254\u201347263","journal-title":"J Biol Chem"},{"issue":"2","key":"50_CR83","doi-asserted-by":"publisher","first-page":"134","DOI":"10.1016\/j.bcmd.2012.09.006","volume":"50","author":"A Zimran","year":"2013","unstructured":"Zimran A, Altarescu G, Elstein D (2013) Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease. Blood Cells Mol Dis 50(2):134\u2013137","journal-title":"Blood Cells Mol Dis"}],"container-title":["Handbook of Experimental Pharmacology","Targeting Trafficking in Drug Development"],"original-title":[],"link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/164_2017_50","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,6,25]],"date-time":"2025-06-25T21:25:38Z","timestamp":1750886738000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/164_2017_50"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017]]},"ISBN":["9783319741635","9783319741642"],"references-count":83,"URL":"https:\/\/doi.org\/10.1007\/164_2017_50","relation":{},"ISSN":["0171-2004","1865-0325"],"issn-type":[{"value":"0171-2004","type":"print"},{"value":"1865-0325","type":"electronic"}],"subject":[],"published":{"date-parts":[[2017]]}}}