Aquaporin 2 Mutations in Nephrogenic Diabetes Insipidus

Seminars in Nephrology - Tập 28 Số 3 - Trang 252-265 - 2008
Anne J. M. Loonen1, Nine V.A.M. Knoers2, C.H. van Os1, Peter M.T. Deen1
1Department of Physiology, Nijmegen Centre for Molecular Life Sciences, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands
2Department of Human Genetics, Nijmegen Centre for Molecular Life Sciences, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands

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King, 1996, Pathophysiology of the aquaporin water channels, Annu Rev Physiol, 58, 619, 10.1146/annurev.ph.58.030196.003155

Koyama, 1998, Cloning and functional expression of human aquaporin8 cDNA and analysis of its gene, Genomics, 54, 169, 10.1006/geno.1998.5552

Borgnia, 1999, Cellular and molecular biology of the aquaporin water channels, Annu Rev Biochem, 68, 425, 10.1146/annurev.biochem.68.1.425

Hara-chikuma, 2006, Physiological roles of glycerol-transporting aquaporins: the aquaglyceroporins, Cell Mol Life Sci, 63, 1386, 10.1007/s00018-006-6028-4

Knepper, 1997, Regulation of aquaporin-2 water channel trafficking by vasopressin, Curr Opin Cell Biol, 9, 560, 10.1016/S0955-0674(97)80034-8

Nonoguchi, 1995, Immunohistochemical localization of V2 vasopressin receptor along the nephron and functional role of luminal V2 receptor in terminal inner medullary collecting ducts, J Clin Invest, 96, 1768, 10.1172/JCI118222

Fushimi, 1997, Phosphorylation of serine 256 is required for cAMP- dependent regulatory exocytosis of the aquaporin-2 water channel, J Biol Chem, 272, 14800, 10.1074/jbc.272.23.14800

Katsura, 1997, Protein kinase A phosphorylation is involved in regulated exocytosis of aquaporin-2 in transfected LLC-PK1 cells, Am J Physiol, 41, F816

Ishibashi, 1997, Immunolocalization and effect of dehydration on AQP3, a basolateral water channel of kidney collecting ducts, Am J Physiol, 41, F235

Kim, 2005, Decreased expression of AQP2 and AQP4 water channels and Na,K-ATPase in kidney collecting duct in AQP3 null mice, Biol Cell, 97, 765, 10.1042/BC20040148

Nielsen, 1995, Vasopressin increases water permeability of kidney collecting duct by inducing translocation of aquaporin-CD water channels to plasma membrane, Proc Natl Acad Sci U S A, 92, 1013, 10.1073/pnas.92.4.1013

Lande, 1996, Phosphorylation of aquaporin-2 does not alter the membrane water permeability of rat papillary water channel- containing vesicles, J Biol Chem, 271, 5552, 10.1074/jbc.271.10.5552

Kamsteeg, 1999, An impaired routing of wild-type aquaporin-2 after tetramerization with an aquaporin-2 mutant explains dominant nephrogenic diabetes insipidus, EMBO J, 18, 2394, 10.1093/emboj/18.9.2394

Van Balkom, 2002, The role of putative phosphorylation sites in the targeting and shuttling of the aquaporin-2 water channel, J Biol Chem, 277, 41473, 10.1074/jbc.M207525200

Christensen, 2000, Localization and regulation of PKA-phosphorylated AQP2 in response to V(2)-receptor agonist/antagonist treatment, Am J Physiol Renal Physiol, 278, F29, 10.1152/ajprenal.2000.278.1.F29

Werten, 2001, Large-scale purification of functional recombinant human aquaporin-2, FEBS Lett, 504, 200, 10.1016/S0014-5793(01)02703-X

Kamsteeg, 2000, The subcellular localization of an aquaporin-2 tetramer depends on the stoichiometry of phosphorylated and nonphosphorylated monomers, J Cell Biol, 151, 919, 10.1083/jcb.151.4.919

Brown, 1998, Cellular mechanisms of aquaporin trafficking, Am J Physiol, 275, F328

Klussmann, 2000, The mechanisms of aquaporin control in the renal collecting duct, Rev Physiol Biochem Pharmacol, 141, 33, 10.1007/BFb0119577

Marples, 1995, Redistribution of aquaporin-2 water channels induced by vasopressin in rat kidney inner medullary collecting duct, Am J Physiol, 38, C655, 10.1152/ajpcell.1995.269.3.C655

Nielsen, 1999, Physiology and pathophysiology of renal aquaporins, J Am Soc Nephrol, 10, 647, 10.1681/ASN.V103647

Katsura, 1996, Direct demonstration of aquaporin-2 water channel recycling in stably transfected LLC-PK1 epithelial cells, Am J Physiol, 39, F548

Digiovanni, 1994, Regulation of collecting duct water channel expression by vasopressin in Brattleboro rat, Proc Natl Acad Sci U S A, 91, 8984, 10.1073/pnas.91.19.8984

van Os, 1994, Aquaporins: water selective channels in biological membranes, Biochim Biophys Acta, 1197, 291, 10.1016/0167-4781(94)00010-Z

Hozawa, 1996, cAMP motifs regulating transcription in the aquaporin 2 gene, Am J Physiol, 39, C1695, 10.1152/ajpcell.1996.270.6.C1695

Yasui, 1997, Adenylate cyclase-coupled vasopressin receptor activates AQP2 promoter via a dual effect on CRE and AP1 elements, Am J Physiol, 41, F443

Matsumura, 1997, Transcriptional regulation of aquaporin-2 water channel gene by cAMP, J Am Soc Nephrol, 8, 861, 10.1681/ASN.V86861

Albertazzi, 2000, Nephrogenic diabetes insipidus: functional analysis of new AVPR2 mutations identified in Italian families, J Am Soc Nephrol, 11, 1033, 10.1681/ASN.V1161033

Arthus, 2000, Report of 33 novel AVPR2 mutations and analysis of 117 families with X- linked nephrogenic diabetes insipidus, J Am Soc Nephrol, 11, 1044, 10.1681/ASN.V1161044

Hansen, 1997, Genetic basis of familial neurohypophyseal diabetes insipidus, Trends Endocrinol Metab, 8, 363, 10.1016/S1043-2760(97)00157-4

Rittig, 2002, Autosomal dominant neurohypophyseal diabetes insipidus due to substitution of histidine for tyrosine(2) in the vasopressin moiety of the hormone precursor, J Clin Endocrinol Metab, 87, 3351, 10.1210/jc.87.7.3351

Robertson, 1995, Pathophysiology of water metabolism, 873

Cipriani, 2005, Lithium in the prevention of suicidal behavior and all-cause mortality in patients with mood disorders: a systematic review of randomized trials, Am J Psychiatry, 162, 1805, 10.1176/appi.ajp.162.10.1805

Stone, 1999, Lithium-induced nephrogenic diabetes insipidus, J Am Board Fam Pract, 12, 43, 10.3122/15572625-12-1-43

Schrier, 2001, Water retention and aquaporins in heart failure, liver disease and pregnancy, J R Soc Med, 94, 265, 10.1177/014107680109400603

Marples, 1999, Long-term regulation of aquaporins in the kidney, Am J Physiol, 276, F331

Deen, 1994, Assignment of the human gene for the water channel of renal collecting duct aquaporin 2 (AQP2) to chromosome 12 region q12-- >q13, Cytogenet Cell Genet, 66, 260, 10.1159/000133707

Sasaki, 1994, Cloning, characterization, and chromosomal mapping of human aquaporin of collecting duct, J Clin Invest, 93, 1250, 10.1172/JCI117079

Mannucci, 1977, 1-Deamino-8-d-arginine vasopressin: a new pharmacological approach to the management of haemophilia and von Willebrands' diseases, Lancet, 1, 869, 10.1016/S0140-6736(77)91197-7

Kaufmann, 2000, Vasopressin-induced von Willebrand factor secretion from endothelial cells involves V2 receptors and cAMP, J Clin Invest, 106, 107, 10.1172/JCI9516

Kobrinsky, 1985, Absent factor VIII response to synthetic vasopressin analogue (DDAVP) in nephrogenic diabetes insipidus, Lancet, 1, 1293, 10.1016/S0140-6736(85)92790-4

Bichet, 1988, Hemodynamic and coagulation responses to 1-desamino[8-D-arginine] vasopressin in patients with congenital nephrogenic diabetes insipidus, N Engl J Med, 318, 881, 10.1056/NEJM198804073181403

Knoers, 1990, Fibrinolytic responses to 1-desamino-8-D-arginine-vasopressin in patients with congenital nephrogenic diabetes insipidus, Nephron, 54, 322, 10.1159/000185888

Knoers, 1991, A variant of nephrogenic diabetes insipidus: V2 receptor abnormality restricted to the kidney, Eur J Pediatr, 150, 370, 10.1007/BF01955943

Fushimi, 1993, Cloning and expression of apical membrane water channel of rat kidney collecting tubule, Nature, 361, 549, 10.1038/361549a0

Saito, 1995, Human AQP2 and MIP genes, two members of the MIP family, map within chromosome band 12q13 on the basis of two-color FISH, Cytogenet Cell Genet, 68, 45, 10.1159/000133885

Deen, 1994, Requirement of human renal water channel aquaporin-2 for vasopressin-dependent concentration of urine, Science, 264, 92, 10.1126/science.8140421

van Lieburg, 1994, Patients with autosomal nephrogenic diabetes insipidus homozygous for mutations in the aquaporin 2 water-channel gene, Am J Hum Genet, 55, 648

van Lieburg, 1996, Normal fibrinolytic responses to 1-desamino-8-D-arginine vasopressin in patients with nephrogenic diabetes insipidus caused by mutations in the aquaporin 2 gene, Nephron, 72, 544, 10.1159/000188936

van Lieburg, 1995, Clinical phenotype of nephrogenic diabetes insipidus in females heterozygous for a vasopressin type 2 receptor mutation, Hum Genet, 96, 70, 10.1007/BF00214189

Moses, 1995, Proposed cause of marked vasopressin resistance in a female with an X-linked recessive V2 receptor abnormality, J Clin Endocrinol Metab, 80, 1184, 10.1210/jc.80.4.1184

Brown, 1990, Differential methylation of the hypervariable locus DXS255 on active and inactive X chromosomes correlates with the expression of a human X-linked gene, Genomics, 7, 215, 10.1016/0888-7543(90)90543-4

Deen, 1995, Water channels encoded by mutant aquaporin-2 genes in nephrogenic diabetes insipidus are impaired in their cellular routing, J Clin Invest, 95, 2291, 10.1172/JCI117920

Marr, 2002, Cell-biologic and functional analyses of five new aquaporin-2 missense mutations that cause recessive nephrogenic diabetes insipidus, J Am Soc Nephrol, 13, 2267, 10.1097/01.ASN.0000027355.41663.14

Marr, 2001, Functionality of aquaporin-2 missense mutants in recessive nephrogenic diabetes insipidus, Pflugers Arch, 442, 73, 10.1007/s004240000498

Mulders, 1997, New mutations in the AQP2 gene in nephrogenic diabetes insipidus resulting in functional but misrouted water channels, J Am Soc Nephrol, 8, 242, 10.1681/ASN.V82242

Tamarappoo, 1998, Defective aquaporin-2 trafficking in nephrogenic diabetes insipidus and correction by chemical chaperones, J Clin Invest, 101, 2257, 10.1172/JCI2303

Kanno, 1995, Urinary excretion of aquaporin-2 in patients with diabetes insipidus, N Engl J Med, 332, 1540, 10.1056/NEJM199506083322303

Yang, 2000, Neonatal mortality in an aquaporin-2 knock-in mouse model of recessive nephrogenic diabetes insipidus, J Biol Chem, 276, 2775, 10.1074/jbc.M008216200

Kamsteeg, 2000, Importance of aquaporin-2 expression levels in genotype -phenotype studies in nephrogenic diabetes insipidus, Am J Physiol Renal Physiol, 279, F778, 10.1152/ajprenal.2000.279.4.F778

Canfield, 1997, Identification and characterization of aquaporin-2 water channel mutations causing nephrogenic diabetes insipidus with partial vasopressin response, Hum Mol Genet, 6, 1865, 10.1093/hmg/6.11.1865

Boccalandro, 2004, Characterization of an aquaporin-2 water channel gene mutation causing partial nephrogenic diabetes insipidus in a Mexican family: evidence of increased frequency of the mutation in the town of origin, J Am Soc Nephrol, 15, 1223, 10.1097/01.ASN.0000125248.85135.43

De Mattia, 2004, A novel mechanism in recessive nephrogenic diabetes insipidus: wild-type aquaporin-2 rescues the apical membrane expression of intracellularly retained AQP2-P262L, Hum Mol Genet, 13, 3045, 10.1093/hmg/ddh339

Kamsteeg, 2003, Reversed polarized delivery of an aquaporin-2 mutant causes dominant nephrogenic diabetes insipidus, J Cell Biol, 163, 1099, 10.1083/jcb.200309017

Kuwahara, 2001, Three families with autosomal dominant nephrogenic diabetes insipidus caused by aquaporin-2 mutations in the C-terminus, Am J Hum Genet, 69, 738, 10.1086/323643

Marr, 2002, Heteroligomerization of an aquaporin-2 mutant with wild-type aquaporin- 2 and their misrouting to late endosomes/lysosomes explains dominant nephrogenic diabetes insipidus, Hum Mol Genet, 11, 779, 10.1093/hmg/11.7.779

Mulders, 1998, An aquaporin-2 water channel mutant which causes autosomal dominant nephrogenic diabetes insipidus is retained in the Golgi complex, J Clin Invest, 102, 57, 10.1172/JCI2605

Afzal, 2000, Recessive Robinow syndrome, allelic to dominant brachydactyly type B, is caused by mutation of ROR2, Nat Genet, 25, 419, 10.1038/78107

Vikkula, 1995, Autosomal dominant and recessive osteochondrodysplasias associated with the COL11A2 locus, Cell, 80, 431, 10.1016/0092-8674(95)90493-X

Wei, 2000, Novel KCNQ1 mutations associated with recessive and dominant congenital long QT syndromes: evidence for variable hearing phenotype associated with R518X, Hum Mutat, 15, 387, 10.1002/(SICI)1098-1004(200004)15:4<387::AID-HUMU26>3.0.CO;2-T

Zhang, 1996, Mutations in the human skeletal muscle chloride channel gene (CLCN1) associated with dominant and recessive myotonia congenita, Neurology, 47, 993, 10.1212/WNL.47.4.993

Deen, 1997, Aquaporin-2 transfection of Madin-Darby canine kidney cells reconstitutes vasopressin-regulated transcellular osmotic water transport, J Am Soc Nephrol, 8, 1493, 10.1681/ASN.V8101493

Procino, 2003, Ser-256 phosphorylation dynamics of aquaporin 2 during maturation from the ER to the vesicular compartment in renal cells, FASEB J, 17, 1886, 10.1096/fj.02-0870fje

Hirano, 2002, Expression of a mutant ER-retained polytope membrane protein in cultured rat hepatocytes results in Mallory body formation, Histochem Cell Biol, 117, 41, 10.1007/s00418-001-0370-2

Asai, 2003, Pathogenesis of nephrogenic diabetes insipidus by aquaporin-2 C-terminus mutations, Kidney Int, 64, 2, 10.1046/j.1523-1755.2003.00049.x

Heilker, 1999, Recognition of sorting signals by clathrin adaptors, Bioessays, 21, 558, 10.1002/(SICI)1521-1878(199907)21:7<558::AID-BIES4>3.0.CO;2-R

Kamsteeg, 2007, Missorting of the aquaporin-2 mutant E258K to multivesicular bodies/lysosomes in dominant NDI is associated with its monoubiquitination and increased phosphorylation by PKC but is due to the loss of E258, Pflugers Arch, 455, 1041, 10.1007/s00424-007-0364-6

Kamsteeg, 2006, Short-chain ubiquitination mediates the regulated endocytosis of the aquaporin-2 water channel, Proc Natl Acad Sci U S A, 103, 18344, 10.1073/pnas.0604073103

De Mattia, 2005, Lack of arginine vasopressin-induced phosphorylation of aquaporin-2 mutant AQP2-R254L explains dominant nephrogenic diabetes insipidus, J Am Soc Nephrol, 16, 2872, 10.1681/ASN.2005010104

Rojek, 2006, Severe urinary concentrating defect in renal collecting duct-selective AQP2 conditional-knockout mice, Proc Natl Acad Sci U S A, 103, 6037, 10.1073/pnas.0511324103

Lloyd, 2005, Diabetes insipidus in mice with a mutation in aquaporin-2, PLoS Genet, 1, e20, 10.1371/journal.pgen.0010020

Sohara, 2006, Pathogenesis and treatment of autosomal-dominant nephrogenic diabetes insipidus caused by an aquaporin 2 mutation, Proc Natl Acad Sci U S A, 103, 14217, 10.1073/pnas.0602331103

McDill, 2006, Congenital progressive hydronephrosis (cph) is caused by an S256L mutation in aquaporin-2 that affects its phosphorylation and apical membrane accumulation, Proc Natl Acad Sci U S A, 103, 6952, 10.1073/pnas.0602087103

Shi, 2007, Nephrogenic diabetes insipidus in mice caused by deleting COOH-terminal tail of aquaporin-2, Am J Physiol Renal Physiol, 292, F1334, 10.1152/ajprenal.00308.2006

Zender, 1992, Traumatic rupture of the urinary tract in a patient presenting nephrogenic diabetes insipidus associated with hydronephrosis and chronic renal failure: case report and review of the literature, Clin Nephrol, 38, 196

Deen, 2007, Mouse models for congenital nephrogenic diabetes insipidus: what can we learn from them?, Nephrol Dial Transplant, 22, 1023, 10.1093/ndt/gfl787

Earley LE, 1961, The effects of infusions of chlorothiazide on urinary dilution and concentration in the dog, J Clin Invest, 40, 857, 10.1172/JCI104320

Kirchlechner, 1999, Treatment of nephrogenic diabetes insipidus with hydrochlorothiazide and amiloride, Arch Dis Child, 80, 548, 10.1136/adc.80.6.548

Alon, 1985, Hydrochlorothiazide-amiloride in the treatment of congenital nephrogenic diabetes insipidus, Am J Nephrol, 5, 9, 10.1159/000166896

Jakobsson, 1994, Effect of hydrochlorothiazide and indomethacin treatment on renal function in nephrogenic diabetes insipidus, Acta Paediatr, 83, 522, 10.1111/j.1651-2227.1994.tb13072.x

Konoshita, 2004, Treatment of congenital nephrogenic diabetes insipidus with hydrochlorothiazide and amiloride in an adult patient, Horm Res, 61, 63, 10.1159/000075241

Rascher, 1987, Congenital nephrogenic diabetes insipidus-vasopressin and prostaglandins in response to treatment with hydrochlorothiazide and indomethacin, Pediatr Nephrol, 1, 485, 10.1007/BF00849258

Morello, 2000, Pharmacological chaperones rescue cell-surface expression and function of misfolded V2 vasopressin receptor mutants, J Clin Invest, 105, 887, 10.1172/JCI8688

Bernier, 2004, Functional rescue of the constitutively internalized V2 vasopressin receptor mutant R137H by the pharmacological chaperone action of SR49059, Mol Endocrinol, 18, 2074, 10.1210/me.2004-0080

Robben, 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, F253, 10.1152/ajprenal.00247.2006

Robben, 2006, Cell biological aspects of the vasopressin type-2 receptor and aquaporin 2 water channel in nephrogenic diabetes insipidus, Am J Physiol Renal Physiol, 291, F257, 10.1152/ajprenal.00491.2005

Hirano, 2003, The proteasome is involved in the degradation of different aquaporin-2 mutants causing nephrogenic diabetes insipidus, Am J Pathol, 163, 111, 10.1016/S0002-9440(10)63635-8

Lin, 2002, Two novel aquaporin-2 mutations responsible for congenital nephrogenic diabetes insipidus in Chinese families, J Clin Endocrinol Metab, 87, 2694, 10.1210/jc.87.6.2694

Tajima, 2003, Two novel aquaporin-2 mutations in a sporadic Japanese patient with autosomal recessive nephrogenic diabetes insipidus, Endocr J, 50, 473, 10.1507/endocrj.50.473

Vargas-Poussou, 1998, Mutations in the vasopressin V2 receptor and aquaporin-2 genes in twelve families with congenital nephrogenic diabetes insipidus, Adv Exp Med Biol, 449, 387, 10.1007/978-1-4615-4871-3_49

Carroll, 2006, Novel mutations underlying nephrogenic diabetes insipidus in Arab families, Genet Med, 8, 443, 10.1097/01.gim.0000223554.46981.7a

Hochberg, 1997, Autosomal recessive nephrogenic diabetes insipidus caused by an aquaporin-2 mutation, J Clin Endocrinol Metab, 82, 686, 10.1210/jc.82.2.686

Goji, 1998, Novel mutations in aquaporin-2 gene in female siblings with nephrogenic diabetes insipidus: evidence of disrupted water channel function, J Clin Endocrinol Metab, 83, 3205, 10.1210/jc.83.9.3205

Kuwahara, 1998, Aquaporin-2, a vasopressin-sensitive water channel, and nephrogenic diabetes insipidus, Intern Med, 37, 215, 10.2169/internalmedicine.37.215

Li, 2003, Altered expression of major renal Na transporters in rats with unilateral ureteral obstruction, Am J Physiol Renal Physiol, 284, F155, 10.1152/ajprenal.00272.2002

Guyon C, Bissonnette P, Lussier Y, et al. Novel aquaporin-2 (AQP2) mutations responsible for autosomal recessive nephrogenic diabetes insipidus [abstract]. J Am Soc Nephrol. 2004.

Iolascon, 2007, Characterization of two novel missense mutations in the AQP2 gene causing nephrogenic diabetes insipidus, Nephron Physiol, 105, 33, 10.1159/000098136

Moses, 1984, Marked hypotonic polyuria resulting from nephrogenic diabetes insipidus with partial sensitivity to vasopressin, J Clin Endocrinol Metab, 59, 1044, 10.1210/jcem-59-6-1044

Oksche, 1996, Two novel mutations in the aquaporin-2 and the vasopressin V2 receptor genes in patients with congenital nephrogenic diabetes insipidus, Hum Genet, 98, 587, 10.1007/s004390050264