Key result
Nine pathogenic missense mutations of ClC-5 exhibited distinct cellular mechanisms of loss of function in Dent disease, including improper N-glycosylation, delayed processing, and reduced currents.
The study identifies distinct cellular mechanisms, including delayed processing and reduced stability, accounting for ClC-5 loss of function in Dent disease.
Mutations in the electrogenic Cl(-)/H(+) exchanger ClC-5 gene CLCN5 are frequently associated with Dent disease, an X-linked recessive disorder affecting the proximal tubules. Here, we investigate the consequences in Xenopus laevis oocytes and in HEK293 cells of nine previously reported, pathogenic, missense mutations of ClC-5, most of them which are located in regions forming the subunit interface. Two mutants trafficked normally to the cell surface and to early endosomes, and displayed complex glycosylation at the cell surface like wild-type ClC-5, but exhibited reduced currents. Three mutants displayed improper N-glycosylation, and were nonfunctional due to being retained and degraded at the endoplasmic reticulum. Functional characterization of four mutants allowed us to identify a novel mechanism leading to ClC-5 dysfunction in Dent disease. We report that these mutant proteins were delayed in their processing, and that the stability of their complex glycosylated form was reduced, causing lower cell surface expression. The early endosome distribution of these mutants was normal. Half of these mutants displayed reduced currents, whereas the other half showed abolished currents. Our study revealed distinct cellular mechanisms accounting for ClC-5 loss of function in Dent disease.
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Grand et al. (2011) studied Dent disease. Pathogenic missense mutations of ClC-5 vs. Wild-type ClC-5 was evaluated on Cellular processing, trafficking, and functional currents of ClC-5 mutants. Nine pathogenic missense mutations of ClC-5 exhibited distinct cellular mechanisms of loss of function in Dent disease, including improper N-glycosylation, delayed processing, and reduced currents.
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