Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemia

Abstract Deletion of exon 9 from Cullin‐3 (CUL3, residues 403–459: CUL3Δ403–459) causes pseudohypoaldosteronism type IIE (PHA2E), a severe form of familial hyperkalaemia and hypertension (FHHt). CUL3 binds the RING protein RBX1 and various substrate adaptors to form Cullin‐RING‐ubiquitin‐ligase comp...

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Main Authors: Frances‐Rose Schumacher, Keith Siew, Jinwei Zhang, Clare Johnson, Nicola Wood, Sarah E Cleary, Raya S Al Maskari, James T Ferryman, Iris Hardege, Yasmin, Nichola L Figg, Radoslav Enchev, Axel Knebel, Kevin M O'Shaughnessy, Thimo Kurz
Format: Article
Language:English
Published: Springer Nature 2015-08-01
Series:EMBO Molecular Medicine
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Online Access:https://doi.org/10.15252/emmm.201505444
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author Frances‐Rose Schumacher
Keith Siew
Jinwei Zhang
Clare Johnson
Nicola Wood
Sarah E Cleary
Raya S Al Maskari
James T Ferryman
Iris Hardege
Yasmin
Nichola L Figg
Radoslav Enchev
Axel Knebel
Kevin M O'Shaughnessy
Thimo Kurz
author_facet Frances‐Rose Schumacher
Keith Siew
Jinwei Zhang
Clare Johnson
Nicola Wood
Sarah E Cleary
Raya S Al Maskari
James T Ferryman
Iris Hardege
Yasmin
Nichola L Figg
Radoslav Enchev
Axel Knebel
Kevin M O'Shaughnessy
Thimo Kurz
author_sort Frances‐Rose Schumacher
collection DOAJ
description Abstract Deletion of exon 9 from Cullin‐3 (CUL3, residues 403–459: CUL3Δ403–459) causes pseudohypoaldosteronism type IIE (PHA2E), a severe form of familial hyperkalaemia and hypertension (FHHt). CUL3 binds the RING protein RBX1 and various substrate adaptors to form Cullin‐RING‐ubiquitin‐ligase complexes. Bound to KLHL3, CUL3‐RBX1 ubiquitylates WNK kinases, promoting their ubiquitin‐mediated proteasomal degradation. Since WNK kinases activate Na/Cl co‐transporters to promote salt retention, CUL3 regulates blood pressure. Mutations in both KLHL3 and WNK kinases cause PHA2 by disrupting Cullin‐RING‐ligase formation. We report here that the PHA2E mutant, CUL3Δ403–459, is severely compromised in its ability to ubiquitylate WNKs, possibly due to altered structural flexibility. Instead, CUL3Δ403–459 auto‐ubiquitylates and loses interaction with two important Cullin regulators: the COP9‐signalosome and CAND1. A novel knock‐in mouse model of CUL3WT/Δ403–459 closely recapitulates the human PHA2E phenotype. These mice also show changes in the arterial pulse waveform, suggesting a vascular contribution to their hypertension not reported in previous FHHt models. These findings may explain the severity of the FHHt phenotype caused by CUL3 mutations compared to those reported in KLHL3 or WNK kinases.
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spelling doaj-art-b8eca7c013e14608b210eecae1e9004c2025-08-20T03:46:16ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842015-08-017101285130610.15252/emmm.201505444Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemiaFrances‐Rose Schumacher0Keith Siew1Jinwei Zhang2Clare Johnson3Nicola Wood4Sarah E Cleary5Raya S Al Maskari6James T Ferryman7Iris Hardege8Yasmin9Nichola L Figg10Radoslav Enchev11Axel Knebel12Kevin M O'Shaughnessy13Thimo Kurz14MRC Protein Phosphorylation and Ubiquitylation Unit, College of Life Sciences, University of DundeeDivision of Experimental Medicine and Immunotherapeutics, University of CambridgeMRC Protein Phosphorylation and Ubiquitylation Unit, College of Life Sciences, University of DundeeMRC Protein Phosphorylation and Ubiquitylation Unit, College of Life Sciences, University of DundeeMRC Protein Phosphorylation and Ubiquitylation Unit, College of Life Sciences, University of DundeeDivision of Experimental Medicine and Immunotherapeutics, University of CambridgeDivision of Experimental Medicine and Immunotherapeutics, University of CambridgeDivision of Experimental Medicine and Immunotherapeutics, University of CambridgeDivision of Experimental Medicine and Immunotherapeutics, University of CambridgeDivision of Experimental Medicine and Immunotherapeutics, University of CambridgeDivision of Cardiovascular Medicine, Department of Medicine, University of CambridgeInstitute of Biochemistry, ETH ZürichMRC Protein Phosphorylation and Ubiquitylation Unit, College of Life Sciences, University of DundeeDivision of Experimental Medicine and Immunotherapeutics, University of CambridgeMRC Protein Phosphorylation and Ubiquitylation Unit, College of Life Sciences, University of DundeeAbstract Deletion of exon 9 from Cullin‐3 (CUL3, residues 403–459: CUL3Δ403–459) causes pseudohypoaldosteronism type IIE (PHA2E), a severe form of familial hyperkalaemia and hypertension (FHHt). CUL3 binds the RING protein RBX1 and various substrate adaptors to form Cullin‐RING‐ubiquitin‐ligase complexes. Bound to KLHL3, CUL3‐RBX1 ubiquitylates WNK kinases, promoting their ubiquitin‐mediated proteasomal degradation. Since WNK kinases activate Na/Cl co‐transporters to promote salt retention, CUL3 regulates blood pressure. Mutations in both KLHL3 and WNK kinases cause PHA2 by disrupting Cullin‐RING‐ligase formation. We report here that the PHA2E mutant, CUL3Δ403–459, is severely compromised in its ability to ubiquitylate WNKs, possibly due to altered structural flexibility. Instead, CUL3Δ403–459 auto‐ubiquitylates and loses interaction with two important Cullin regulators: the COP9‐signalosome and CAND1. A novel knock‐in mouse model of CUL3WT/Δ403–459 closely recapitulates the human PHA2E phenotype. These mice also show changes in the arterial pulse waveform, suggesting a vascular contribution to their hypertension not reported in previous FHHt models. These findings may explain the severity of the FHHt phenotype caused by CUL3 mutations compared to those reported in KLHL3 or WNK kinases.https://doi.org/10.15252/emmm.201505444cullinCUL3monogenic hypertension syndromesproteasomeubiquitinWNK/SPAK/OSR1 pathway
spellingShingle Frances‐Rose Schumacher
Keith Siew
Jinwei Zhang
Clare Johnson
Nicola Wood
Sarah E Cleary
Raya S Al Maskari
James T Ferryman
Iris Hardege
Yasmin
Nichola L Figg
Radoslav Enchev
Axel Knebel
Kevin M O'Shaughnessy
Thimo Kurz
Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemia
EMBO Molecular Medicine
cullin
CUL3
monogenic hypertension syndromes
proteasome
ubiquitin
WNK/SPAK/OSR1 pathway
title Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemia
title_full Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemia
title_fullStr Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemia
title_full_unstemmed Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemia
title_short Characterisation of the Cullin‐3 mutation that causes a severe form of familial hypertension and hyperkalaemia
title_sort characterisation of the cullin 3 mutation that causes a severe form of familial hypertension and hyperkalaemia
topic cullin
CUL3
monogenic hypertension syndromes
proteasome
ubiquitin
WNK/SPAK/OSR1 pathway
url https://doi.org/10.15252/emmm.201505444
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