Novel targets for the treatment of autosomal dominant polycystic kidney disease

Bibliography

• Chapman AB, Guay-Woodford LM, Grantham JJ, Renal structure in early autosomal-dominant polycystic kidney disease (ADPKD): The Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP) cohort. Kidney Int 2003;64:1035-45
   PubMed Web of Science ®Google Scholar
• Grantham JJ, Chapman AB, Torres VE. Volume progression in autosomal dominant polycystic kidney disease: the major factor determining clinical outcomes. Clin J Am Soc Nephrol 2006;1:148-57
   PubMed Web of Science ®Google Scholar
• Torres VE, Harris PC. Autosomal dominant polycystic kidney disease: the last 3 years. Kidney Int 2009;76(2):149-68
   PubMed Web of Science ®Google Scholar
• Rossetti S, Harris PC. Genotype-phenotype correlations in autosomal dominant and autosomal recessive polycystic kidney disease. J Am Soc Nephrol 2007;18(5):1374-80
   PubMed Web of Science ®Google Scholar
• Harris PC. Autosomal dominant polycystic kidney disease: clues to pathogenesis. Hum Mol Genet 1999;8:1861-186
   PubMed Web of Science ®Google Scholar
• Davenport JR, Watts AJ, Roper VC, Disruption of intraflagellar transport in adult mice leads to obesity and slow-onset cystic kidney disease. Curr Biol 2007;17(18):1586-94
   PubMed Web of Science ®Google Scholar
• Jiang ST, Chiou YY, Wang E, Defining a link with autosomal-dominant polycystic kidney disease in mice with congenitally low expression of Pkd1. Am J Pathol 2006;168(1):205-20
   PubMed Web of Science ®Google Scholar
• Thivierge C, Kurbegovic A, Couillard M, Overexpression of PKD1 causes polycystic kidney disease. Mol Cell Biol 2006;26(4):1538-48
   PubMed Web of Science ®Google Scholar
• Park EY, Sung YH, Yang MH, Cyst formation in kidney via B-Raf signaling in the PKD2 transgenic mice. J Biol Chem 2009;284(11):7214-22
   PubMed Web of Science ®Google Scholar
• Geng L, Segal Y, Peissel B, Identification and localization of polycystin, the PKD1 gene product. J Clin Invest 1996;98(12):2674-82
   PubMed Web of Science ®Google Scholar
• Geng L, Segal Y, Pavlova A, Distribution and developmentally regulated expression of murine polycystin. Am J Physiol 1997;272(4 Pt 2):F451-9
   PubMed Web of Science ®Google Scholar
• Huan Y, van Adelsberg J. Polycystin-1, the PKD1 gene product, is in a complex containing E-cadherin and the catenins. J Clin Invest 1999;104(10):1459-68
   PubMed Web of Science ®Google Scholar
• Scheffers MS, van der BP, Prins F, Polycystin-1, the product of the polycystic kidney disease 1 gene, co-localizes with desmosomes in MDCK cells. Hum Mol Genet 2000;9(18):2743-50
   PubMed Web of Science ®Google Scholar
• Tsiokas L, Kim E, Arnould T, Homo- and heterodimeric interactions between the gene products of PKD1 and PKD2. Proc Natl Acad Sci USA 1997;94(13):6965-70
   PubMed Web of Science ®Google Scholar
• Qian F, Germino FJ, Cai Y, PKD1 interacts with PKD2 through a probable coiled-coil domain. Nat Genet 1997;16(2):179-83
   PubMed Web of Science ®Google Scholar
• Nauli SM, Alenghat FJ, Luo Y, Polycystins 1 and 2 mediate mechanosensation in the primary cilium of kidney cells. Nat Genet 2003;33(2):129-37
   PubMed Web of Science ®Google Scholar
• Leuenroth SJ, Okuhara D, Shotwell JD, Triptolide is a traditional Chinese medicine-derived inhibitor of polycystic kidney disease. Proc Natl Acad Sci USA 2007;104:4389-94
   PubMed Web of Science ®Google Scholar
• Leuenroth SJ, Bencivenga N, Igarashi P, Triptolide reduces cystogenesis in a model of ADPKD. J Am Soc Nephrol 2008;19:1659-62
   PubMed Web of Science ®Google Scholar
• Kiviharju TM, Lecane PS, Sellers RG, Peehl DM. Antiproliferative and proapoptotic activities of triptolide (PG490), a natural product entering clinical trials, on primary cultures of human prostatic epithelial cells. Clin Cancer Res 2002;8(8):2666-74
   PubMed Web of Science ®Google Scholar
• Cai Y, Somlo S. Too much of a good thing: does Nek8 link polycystic kidney disease and nephronophthisis? J Am Soc Nephrol 2008;19(3):418-20
   PubMed Web of Science ®Google Scholar
• Sohara E, Luo Y, Zhang J, Nek8 regulates the expression and localization of polycystin-1 and polycystin-2. J Am Soc Nephrol 2008;19(3):469-76
   PubMed Web of Science ®Google Scholar
• Trapp ML, Galtseva A, Manning DK, Defects in ciliary localization of Nek8 is associated with cystogenesis. Pediatr Nephrol 2008;23(3):377-87
   PubMed Web of Science ®Google Scholar
• Valkova N, Yunis R, Mak SK, Nek8 mutation causes overexpression of galectin-1, sorcin, and vimentin and accumulation of the major urinary protein in renal cysts of jck mice. Mol Cell Proteomics 2005;4(7):1009-18
   PubMed Web of Science ®Google Scholar
• Liu S, Lu W, Obara T, A defect in a novel Nek-family kinase causes cystic kidney disease in the mouse and in zebrafish. Development 2002;129(24):5839-46
   PubMed Web of Science ®Google Scholar
• Li X, Luo Y, Starremans PG, Polycystin-1 and polycystin-2 regulate the cell cycle through the helix-loop-helix inhibitor Id2. Nat Cell Biol 2005;7:1102-12
   Web of Science ®Google Scholar
• Schwiebert EM, Wallace DP, Braunstein GM, Autocrine extracellular purinergic signaling in epithelial cells derived from polycystic kidneys. Am J Physiol Renal Physiol 2002;282:F763-75
   PubMed Web of Science ®Google Scholar
• Wallace DP, Rome LA, Sullivan LP, Grantham JJ. cAMP-dependent fluid secretion in rat inner medullary collecting ducts. Am J Physiol Renal Physiol 280:F1019-29
   Google Scholar
• Wallace DP, Christensen M, Reif G, Electrolyte and fluid secretion by cultured human inner medullary collecting duct cells. Am J Physiol Renal Physiol 2002;283:F1337-50
   PubMed Web of Science ®Google Scholar
• Yang B, Sonawane ND, Zhao D, Small-molecule CFTR inhibitors slow cyst growth in polycystic kidney disease. J Am Soc Nephrol 2008;19(7):1300-10
   PubMed Web of Science ®Google Scholar
• Albaqumi M, Srivastava S, Li Z, KCa3.1 potassium channels are critical for cAMP-dependent chloride secretion and cyst growth in autosomal-dominant polycystic kidney disease. Kidney Int 2008;74(6):740-9
   PubMed Web of Science ®Google Scholar
• Alper SL. Let's look at cysts from both sides now. Kidney Int 2008;74(6):699-702
   PubMed Web of Science ®Google Scholar
• Masyuk TV, Masyuk AI, Torres VE, Octreotide inhibits hepatic cystogenesis in a rodent model of polycystic liver disease by reducing cholangiocyte adenosine 3′,5′-cyclic monophosphate. Gastroenterology 2007;132:1104-16
   PubMed Web of Science ®Google Scholar
• Ruggenenti P, Remuzzi A, Ondei P, Safety and efficacy of long-acting somatostatin treatment in autosomal-dominant polycystic kidney disease. Kidney Int 2005;68:206-16
   PubMed Web of Science ®Google Scholar
• Yamaguchi T, Pelling JC, Ramaswamy NT, cAMP stimulates the in vitro proliferation of renal cyst epithelial cells by activating the extracellular signal-regulated kinase pathway. Kidney Int 2000;57:1460-71
   PubMed Web of Science ®Google Scholar
• Yamaguchi T, Wallace DP, Magenheimer BS, Calcium restriction allows cAMP activation of the B-Raf/ERK pathway, switching cells to a cAMP-dependent growth-stimulated phenotype. J Biol Chem 2004;279:40419-30
   PubMed Web of Science ®Google Scholar
• Omori S, Hida M, Fujita H, Extracellular signal-regulated kinase inhibition slows disease progression in mice with polycystic kidney disease. J Am Soc Nephrol 2006;17:1604-14
   PubMed Web of Science ®Google Scholar
• Shibazaki S, Yu Z, Nishio S, Cyst formation and activation of the extracellular regulated kinase pathway after kidney specific inactivation of Pkd1. Hum Mol Genet 2008;17(11):1505-16
   PubMed Web of Science ®Google Scholar
• Parker E, Newby LJ, Sharpe CC, Hyperproliferation of PKD1 cystic cells is induced by insulin-like growth factor-1 activation of the Ras/Raf signalling system. Kidney Int 2007;72:157-65
   PubMed Web of Science ®Google Scholar
• Zhang Y, Gao X, Saucedo LJ, Rheb is a direct target of the tuberous sclerosis tumour suppressor proteins. Nat Cell Biol 2003;5:578-81
   PubMed Web of Science ®Google Scholar
• Torres VE, Wang X, Qian Q, Effective treatment of an orthologous model of autosomal dominant polycystic kidney disease. Nat Med 2004;10:363-4
   PubMed Web of Science ®Google Scholar
• Gattone VH, Wang X, Harris PC, Torres VE. Inhibition of renal cystic disease development and progression by a vasopressin V2 receptor antagonist. Nat Med 2003;9:1323-6
   PubMed Web of Science ®Google Scholar
• Wang X, Gattone V, Harris PC, Torres VE. Effectiveness of vasopressin V2 receptor antagonists OPC-31260 and OPC-41061 on polycystic kidney disease development in the PCK rat. J Am Soc Nephrol 2005;16:846-51
   PubMed Web of Science ®Google Scholar
• Guay-Woodford LM. Murine models of polycystic kidney disease: molecular and therapeutic insights. Am J Physiol Renal Physiol 2003;285:F1034-49
   PubMed Web of Science ®Google Scholar
• Nagao S, Nishii K, Katsuyama M, Increased water intake decreases progression of polycystic kidney disease in the PCK rat. J Am Soc Nephrol 2006;17(8):2220-7
   PubMed Web of Science ®Google Scholar
• Grantham JJ, Chapman AB, Torres VE, Acute and chronic osmostasis after vasopressin V2 receptor inhibition with tolvaptan in ADPKD [abstract]. J Am Soc Nephrol 2005;16:361A
   Google Scholar
• Torres VE, Wang X, Ward CJ, Urine aquaporin -2 and cyclic AMP responses to tolvaptan administration in autosomal dominant kidney disease [abstract]. J Am Soc Nephrol 2005;16:361A
   Google Scholar
• Chapman AB, Torres VE, Grantham JJ, A phase IIB pilot study fof the safety and efficacy of tolvaptan, a vasopressin V2 receptor antagonist (V2RA) in patients with ADPKD [abstract]. J Am Soc Nephrol 2005;16:68A
   PubMed Web of Science ®Google Scholar
• Chapman AB. Autosomal dominant polycystic kidney disease: time for a change? J Am Soc Nephrol 2007;18:1399-407
   PubMed Web of Science ®Google Scholar
• Sweeney WE, Chen Y, Nakanishi K, Treatment of polycystic kidney disease with a novel tyrosine kinase inhibitor. Kidney Int 2000;57:33-40
   PubMed Web of Science ®Google Scholar
• Nakanishi K, Gattone VH, Sweeney WE, Avner ED. Renal dysfunction but not cystic change is ameliorated by neonatal epidermal growth factor in bpk mice. Pediatr Nephrol 2001;16:45-50
   PubMed Web of Science ®Google Scholar
• Sweeney WE, Futey L, Frost P, Avner ED. In vitro modulation of cyst formation by a novel tyrosine kinase inhibitor. Kidney Int 1999;56:406-13
   PubMed Web of Science ®Google Scholar
• Sweeney WE, Jr., Hamahira K, Sweeney J, Combination treatment of PKD utilizing dual inhibition of EGF-receptor activity and ligand bioavailability. Kidney Int 2003;64:1310-19
   PubMed Web of Science ®Google Scholar
• Torres VE, Sweeney WE, Jr., Wang X, Avner ED: EGF receptor tyrosine kinase inhibition attenuates the development of PKD in Han:SPRD rats. Kidney Int 2003;64:1573-9
   PubMed Web of Science ®Google Scholar
• Sweeney WE Jr, von Vigier RO, Frost P, Avner ED. Src inhibition ameliorates polycystic kidney disease. J Am Soc Nephrol 2008;19:1331-41
   PubMed Web of Science ®Google Scholar
• Edelstein CL. Mammalian target of rapamycin and caspase inhibitors in polycystic kidney disease. Clin J Am Soc Nephrol 2008;3:1219-26
   PubMed Web of Science ®Google Scholar
• Fingar DC, Blenis J. Target of rapamycin (TOR): an integrator of nutrient and growth factor signals and coordinator of cell growth and cell cycle progression. Oncogene 2004;23:3151-71
   PubMed Web of Science ®Google Scholar
• Gao X, Zhang Y, Arrazola P, Tsc tumour suppressor proteins antagonize amino-acid-TOR signalling. Nat Cell Biol 2002;4:699-704
   PubMed Web of Science ®Google Scholar
• Hara K, Maruki Y, Long X, Raptor, a binding partner of target of rapamycin (TOR), mediates TOR action. Cell 2002;110:177-89
   PubMed Web of Science ®Google Scholar
• Burnett PE, Barrow RK, Cohen NA, RAFT1 phosphorylation of the translational regulators p70 S6 kinase and 4E-BP1. Proc Natl Acad Sci USA 1998;95:1432-7
   PubMed Web of Science ®Google Scholar
• Oshiro N, Yoshino K, Hidayat S, Dissociation of raptor from mTOR is a mechanism of rapamycin-induced inhibition of mTOR function. Genes Cells 2004;9:359-66. [Erratum appears in Genes Cells 2004;9(5):497]
   PubMed Web of Science ®Google Scholar
• Wahl PR, Serra AL, Le Hir M, Inhibition of mTOR with sirolimus slows disease progression in Han:SPRD rats with autosomal dominant polycystic kidney disease (ADPKD). Nephrol Dial Transplant 2006;21:598-604
   PubMed Web of Science ®Google Scholar
• Shillingford JM, Murcia NS, Larson CH, The mTOR pathway is regulated by polycystin-1, and its inhibition reverses renal cystogenesis in polycystic kidney disease. Proc Natl Acad Sci USA 2006;103:5466-71
   PubMed Web of Science ®Google Scholar
• Wu M, Wahl PR, Le Hir M, Everolimus retards cyst growth and preserves kidney function in a rodent model for polycystic kidney disease. Kidney Blood Press Res 2007;30:253-9
   PubMed Web of Science ®Google Scholar
• Tao Y, Kim J, Schrier RW, Edelstein CL. Rapamycin markedly slows disease progression in a rat model of polycystic kidney disease (PKD). J Am Soc Nephrol 2005;16:46-51
   PubMed Web of Science ®Google Scholar
• Qian Q, Hui D, King BF, Sirolimus reduces polycystic liver volume in ADPKD patients. J Am Soc Nephrol 2008;19:631-8
   PubMed Web of Science ®Google Scholar
• Lee L, Sudentas P, Donohue B, Efficacy of a rapamycin analog (CCI-779) and IFN-gamma in tuberous sclerosis mouse models. Genes Chromosomes Cancer 2005;42:213-27
   PubMed Web of Science ®Google Scholar
• Bissler JJ, McCormack FX, Young LR, Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med 2008;358:140-51
   PubMed Web of Science ®Google Scholar
• Serra AL, Kistler AD, Poster D, Safety and tolerability of sirolimus treatment in patients with autosomal dominant polycystic kidney disease. Nephrol Dial Transplant 2009;24(11):3334-42
   PubMed Web of Science ®Google Scholar
• Nakamura T, Ebihara I, Nagaoka I, Growth factor gene expression in kidney of murine polycystic kidney disease. J Am Soc Nephrol 1993;3:1378-86
   PubMed Web of Science ®Google Scholar
• Aukema HM, Housini I. Dietary soy protein effects on disease and IGF-I in male and female Han:SPRD-cy rats. Kidney Int 2001;59:52-61
   PubMed Web of Science ®Google Scholar
• Mehls O, Irzynjec T, Ritz E, Effects of rhGH and rhIGF-1 on renal growth and morphology. Kidney Int 1993;44:1251-8
   PubMed Web of Science ®Google Scholar
• Du J, Wilson PD. Abnormal polarization of EGF receptors and autocrine stimulation of cyst epithelial growth in human ADPKD. Am J Physiol 1995;269:C487-95
   PubMed Web of Science ®Google Scholar
• Alvaro D, Onori P, Alpini G, Morphological and functional features of hepatic cyst epithelium in autosomal dominant polycystic kidney disease. Am J Pathol 2008;172:321-32
   PubMed Web of Science ®Google Scholar
• Nishio S, Hatano M, Nagata M, Pkd1 regulates immortalized proliferation of renal tubular epithelial cells through p53 induction and JNK activation. J Clin Invest 2005;115:910-18
   PubMed Web of Science ®Google Scholar
• Otto EA, Schermer B, Obara T, Mutations in INVS encoding inversin cause nephronophthisis type 2, linking renal cystic disease to the function of primary cilia and left-right axis determination. Nat Genet 2003;34:413-20
   PubMed Web of Science ®Google Scholar
• Pazour GJ. Intraflagellar transport and cilia-dependent renal disease: the ciliary hypothesis of polycystic kidney disease. J Am Soc Nephrol 2004;15:2528-36
   PubMed Web of Science ®Google Scholar
• Bhunia AK, Piontek K, Boletta A, PKD1 induces p21(waf1) and regulation of the cell cycle via direct activation of the JAK-STAT signaling pathway in a process requiring PKD2. Cell 2002;109:157-68
   PubMed Web of Science ®Google Scholar
• Bukanov NO, Smith LA, Klinger KW, Long lasting arrest of murine polycystic kidney disease with CDK inhibitor roscovitine. Nature 2006;444:949-52
   PubMed Web of Science ®Google Scholar
• MacCallum DE, Melville J, Frame S, Seliciclib (CYC202, R-Roscovitine) induces cell death in multiple myeloma cells by inhibition of RNA polymerase II-dependent transcription and down-regulation of Mcl-1. Cancer Res 2005;65:5399-407
   PubMed Web of Science ®Google Scholar
• McClue SJ, Blake D, Clarke R, In vitro and in vivo antitumor properties of the cyclin dependent kinase inhibitor CYC202 (R-roscovitine). Int J Cancer 2002;102:463-8
   PubMed Web of Science ®Google Scholar
• Song H, Vita M, Sallam H, Effect of the Cdk-inhibitor roscovitine on mouse hematopoietic progenitors in vivo and in vitro. Cancer Chemother Pharmacol 2007;60(6):841-9
   PubMed Web of Science ®Google Scholar
• Li X, Magenheimer BS, Xia S, A tumor necrosis factor-alpha-mediated pathway promoting autosomal dominant polycystic kidney disease. Nat Med 2008;14:863-8
   PubMed Web of Science ®Google Scholar
• Wilson PD. Polycystic kidney disease. N Engl J Med 2004;350:151-64
   PubMed Web of Science ®Google Scholar
• Thomas GL, Yang B, Wagner BE, Cellular apoptosis and proliferation in experimental renal fibrosis. Nephrol Dial Transplant 1998;13:2216-26
   PubMed Web of Science ®Google Scholar
• Cao Z, Cooper ME. Role of angiotensin II in tubulointerstitial injury. Semin Nephrol 2001;21:554-62
   PubMed Web of Science ®Google Scholar
• Schindler R, Dinarello CA, Koch KM. Angiotensin-converting-enzyme inhibitors suppress synthesis of tumour necrosis factor and interleukin 1 by human peripheral blood mononuclear cells. Cytokine 1995;7:526-33
   PubMed Web of Science ®Google Scholar
• Kennefick TM, Al-Nimri MA, Oyama TT, Hypertension and renal injury in experimental polycystic kidney disease. Kidney Int 1999;56:2181-90
   PubMed Web of Science ®Google Scholar
• Keith DS, Torres VE, Johnson CM, Holley KE. Effect of sodium chloride, enalapril, and losartan on the development of polycystic kidney disease in Han:SPRD rats. Am J Kidney Dis 1994;24:491-8
   PubMed Web of Science ®Google Scholar
• Zafar I, Tao Y, Falk S, Effect of statin and angiotensin-converting enzyme inhibition on structural and hemodynamic alterations in autosomal dominant polycystic kidney disease model. Am J Physiol Renal Physiol 2007;293:F854-9
   PubMed Web of Science ®Google Scholar
• Jafar TH, Stark PC, Schmid CH, ; ACE Inhibition in Progressive Renal Disease (AIPRD) Study Group. The effect of angiotensin-converting-enzyme inhibitors on progression of advanced polycystic kidney disease. Kidney Int 2005;67:265-71
   PubMed Web of Science ®Google Scholar
• Ogborn MR, Sareen S, Pinette G. Cilazapril delays progression of hypertension and uremia in rat polycystic kidney disease. Am J Kidney Dis 1995;26:942-6
   PubMed Web of Science ®Google Scholar
• Rubanyi GM. Endothelium-dependent pressure-induced contraction of isolated canine carotid arteries. Am J Physiol 1988;255:H783-8
   PubMed Web of Science ®Google Scholar
• Chapman AB, Johnson A, Gabow PA, Schrier RW. The renin-angiotensin-aldosterone system and autosomal dominant polycystic kidney disease. N Engl J Med 1990;323:1091-6
   PubMed Web of Science ®Google Scholar
• Levey AS, Greene T, Beck GJ, Dietary protein restriction and the progression of chronic renal disease: what have all of the results of the MDRD study shown? Modification of Diet in Renal Disease Study group. J Am Soc Nephrol 1999;10:2426-39
   PubMed Web of Science ®Google Scholar
• Remuzzi G, Bertani T. Pathophysiology of progressive nephropathies. N Engl J Med 1998;339:1448-56
   PubMed Web of Science ®Google Scholar
• Schrier R, McFann K, Johnson A, Cardiac and renal effects of standard versus rigorous blood pressure control in autosomal-dominant polycystic kidney disease: results of a seven-year prospective randomized study. J Am Soc Nephrol 2002;13:1733-9
   PubMed Web of Science ®Google Scholar
• Amico P, Kalbermatter S, Kiss D. Aliskiren corrects recurrent hyperreninemia and hyperaldosteronism in autosomaldominant polycystic kidney disease. Clin Nephrol 2009;72(3):237-9
   PubMed Web of Science ®Google Scholar
• Gile RD, Cowley BD Jr, Gattone VH2, Effect of lovastatin on the development of polycystic kidney disease in the Han:SPRD rat. Am J Kidney Dis 1995;26:501-7
   PubMed Web of Science ®Google Scholar
• van Dijk MA, Kamper AM, van Veen S, Effect of simvastatin on renal function in autosomal dominant polycystic kidney disease. Nephrol Dial Transplant 2001;16:2152-7
   PubMed Web of Science ®Google Scholar
• Namli S, Oflaz H, Turgut F, Improvement of endothelial dysfunction with simvastatin in patients with autosomal dominant polycystic kidney disease. Ren Fail 2007;29:55-9
   PubMed Web of Science ®Google Scholar
• Rankin CA, Itoh Y, Tian C, Matrix metalloproteinase-2 in a murine model of infantile-type polycystic kidney disease. J Am Soc Nephrol 1999;10(2):210-7
   PubMed Web of Science ®Google Scholar
• Rankin CA, Suzuki K, Itoh Y, Matrix metalloproteinases and TIMPS in cultured C57BL/6J-cpk kidney tubules. Kidney Int 1996;50(3):835-44
   PubMed Web of Science ®Google Scholar
• Obermuller N, Morente N, Kranzlin B, A possible role for metalloproteinases in renal cyst development. Am J Physiol Renal Physiol 2001;280(3):F540-50
   PubMed Web of Science ®Google Scholar