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@ARTICLE{Klar:276855,
author = {Klar, Joakim and Piontek, Jörg and Milatz, Susanne and
Tariq, Muhammad and Jameel, Muhammad and Breiderhoff, Tilman
and Schuster, Jens and Fatima, Ambrin and Asif, Maria and
Sher, Muhammad and Mäbert, Katrin and Fromm, Anja and Baig,
Shahid M. and Günzel, Dorothee and Dahl, Niklas},
title = {{A}ltered paracellular cation permeability due to a rare
{CLDN}10{B} variant causes anhidrosis and kidney damage},
journal = {PLoS Genetics},
volume = {13},
number = {7},
issn = {1553-7390},
address = {San Francisco, Calif.},
publisher = {Public Library of Science},
reportid = {DZNE-2025-00368},
pages = {e1006897},
year = {2017},
abstract = {Claudins constitute the major component of tight junctions
and regulate paracellular permeability of epithelia.
Claudin-10 occurs in two major isoforms that form
paracellular channels with ion selectivity. We report on two
families segregating an autosomal recessive disorder
characterized by generalized anhidrosis, severe heat
intolerance and mild kidney failure. All affected
individuals carry a rare homozygous missense mutation
c.144C>G, p.(N48K) specific for the claudin-10b isoform.
Immunostaining of sweat glands from patients suggested that
the disease is associated with reduced levels of claudin-10b
in the plasma membranes and in canaliculi of the secretory
portion. Expression of claudin-10b N48K in a 3D cell model
of sweat secretion indicated perturbed paracellular Na+
transport. Analysis of paracellular permeability revealed
that claudin-10b N48K maintained cation over anion
selectivity but with a reduced general ion conductance.
Furthermore, freeze fracture electron microscopy showed that
claudin-10b N48K was associated with impaired tight junction
strand formation and altered cis-oligomer formation. These
data suggest that claudin-10b N48K causes anhidrosis and our
findings are consistent with a combined effect from
perturbed TJ function and increased degradation of
claudin-10b N48K in the sweat glands. Furthermore, affected
individuals present with Mg2+ retention, secondary
hyperparathyroidism and mild kidney failure that suggest a
disturbed reabsorption of cations in the kidneys. These
renal-derived features recapitulate several phenotypic
aspects detected in mice with kidney specific loss of both
claudin-10 isoforms. Our study adds to the spectrum of
phenotypes caused by tight junction proteins and
demonstrates a pivotal role for claudin-10b in maintaining
paracellular Na+ permeability for sweat production and
kidney function.},
ddc = {610},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
doi = {10.1371/journal.pgen.1006897},
url = {https://pub.dzne.de/record/276855},
}