Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.

Mutations in the SLC4A1 gene encoding the anion exchanger 1 (AE1) can cause distal renal tubular acidosis (dRTA), a disease often due to mis-trafficking of the mutant protein. In this study, we investigated whether trafficking of a Golgi-retained dRTA mutant, G701D kAE1, or two dRTA mutants retained...

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Main Authors: Carmen Y S Chu, Jennifer C King, Mattia Berrini, R Todd Alexander, Emmanuelle Cordat
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2013-01-01
Series:PLoS ONE
Online Access:https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0057062&type=printable
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author Carmen Y S Chu
Jennifer C King
Mattia Berrini
R Todd Alexander
Emmanuelle Cordat
author_facet Carmen Y S Chu
Jennifer C King
Mattia Berrini
R Todd Alexander
Emmanuelle Cordat
author_sort Carmen Y S Chu
collection DOAJ
description Mutations in the SLC4A1 gene encoding the anion exchanger 1 (AE1) can cause distal renal tubular acidosis (dRTA), a disease often due to mis-trafficking of the mutant protein. In this study, we investigated whether trafficking of a Golgi-retained dRTA mutant, G701D kAE1, or two dRTA mutants retained in the endoplasmic reticulum, C479W and R589H kAE1, could be functionally rescued to the plasma membrane of Madin-Darby Canine Kidney (MDCK) cells. Treatments with DMSO, glycerol, the corrector VX-809, or low temperature incubations restored the basolateral trafficking of G701D kAE1 mutant. These treatments had no significant rescuing effect on trafficking of the mis-folded C479W or R589H kAE1 mutants. DMSO was the only treatment that partially restored G701D kAE1 function in the plasma membrane of MDCK cells. Our experiments show that trafficking of intracellularly retained dRTA kAE1 mutants can be partially restored, and that one chemical treatment rescued both trafficking and function of a dRTA mutant. These studies provide an opportunity to develop alternative therapeutic solutions for dRTA patients.
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spelling doaj-art-b4ee0af807f24bfcb181349ecacadd592025-08-20T02:30:38ZengPublic Library of Science (PLoS)PLoS ONE1932-62032013-01-0182e5706210.1371/journal.pone.0057062Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.Carmen Y S ChuJennifer C KingMattia BerriniR Todd AlexanderEmmanuelle CordatMutations in the SLC4A1 gene encoding the anion exchanger 1 (AE1) can cause distal renal tubular acidosis (dRTA), a disease often due to mis-trafficking of the mutant protein. In this study, we investigated whether trafficking of a Golgi-retained dRTA mutant, G701D kAE1, or two dRTA mutants retained in the endoplasmic reticulum, C479W and R589H kAE1, could be functionally rescued to the plasma membrane of Madin-Darby Canine Kidney (MDCK) cells. Treatments with DMSO, glycerol, the corrector VX-809, or low temperature incubations restored the basolateral trafficking of G701D kAE1 mutant. These treatments had no significant rescuing effect on trafficking of the mis-folded C479W or R589H kAE1 mutants. DMSO was the only treatment that partially restored G701D kAE1 function in the plasma membrane of MDCK cells. Our experiments show that trafficking of intracellularly retained dRTA kAE1 mutants can be partially restored, and that one chemical treatment rescued both trafficking and function of a dRTA mutant. These studies provide an opportunity to develop alternative therapeutic solutions for dRTA patients.https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0057062&type=printable
spellingShingle Carmen Y S Chu
Jennifer C King
Mattia Berrini
R Todd Alexander
Emmanuelle Cordat
Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
PLoS ONE
title Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
title_full Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
title_fullStr Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
title_full_unstemmed Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
title_short Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
title_sort functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells
url https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0057062&type=printable
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