Defects in Stratum Corneum Desquamation Are the Predominant Effect of Impaired ABCA12 Function in a Novel Mouse Model of Harlequin Ichthyosis.

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Version: Final published version
Serval ID
serval:BIB_E7BD8871C80D
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Defects in Stratum Corneum Desquamation Are the Predominant Effect of Impaired ABCA12 Function in a Novel Mouse Model of Harlequin Ichthyosis.
Journal
PloS one
Author(s)
Zhang L., Ferreyros M., Feng W., Hupe M., Crumrine D.A., Chen J., Elias P.M., Holleran W.M., Niswander L., Hohl D., Williams T., Torchia E.C., Roop D.R.
ISSN
1932-6203 (Electronic)
ISSN-L
1932-6203
Publication state
Published
Issued date
2016
Peer-reviewed
Oui
Volume
11
Number
8
Pages
e0161465
Language
english
Notes
Publication types: Journal Article
Publication Status: epublish
Abstract
Harlequin Ichthyosis is a severe skin disease caused by mutations in the human gene encoding ABCA12. Here, we characterize a novel mutation in intron 29 of the mouse Abca12 gene that leads to the loss of a 5' splice donor site and truncation of the Abca12 RNA transcript. Homozygous mutants of this smooth skin or smsk allele die perinatally with shiny translucent skin, typical of animal models of Harlequin Ichthyosis. Characterization of smsk mutant skin showed that the delivery of glucosylceramides and CORNEODESMOSIN was defective, while ultrastructural analysis revealed abnormal lamellar bodies and the absence of lipid lamellae in smsk epidermis. Unexpectedly, mutant stratum corneum remained intact when subjected to harsh chemical dissociation procedures. Moreover, both KALLIKREIN 5 and -7 were drastically decreased, with retention of desmoplakin in mutant SC. In cultured wild type keratinocytes, both KALLIKREIN 5 and -7 colocalized with ceramide metabolites following calcium-induced differentiation. Reducing the intracellular levels of glucosylceramide with a glucosylceramide synthase inhibitor resulted in decreased secretion of KALLIKREIN proteases by wild type keratinocytes, but not by smsk mutant keratinocytes. Together, these findings suggest an essential role for ABCA12 in transferring not only lipids, which are required for the formation of multilamellar structures in the stratum corneum, but also proteolytic enzymes that are required for normal desquamation. Smsk mutant mice recapitulate many of the pathological features of HI and can be used to explore novel topical therapies against a potentially lethal and debilitating neonatal disease.

Keywords
ATP-Binding Cassette Transporters/genetics, Alleles, Animals, Base Sequence, Ceramides/metabolism, Chromosome Mapping, Desmosomes/metabolism, Disease Models, Animal, Epidermis/metabolism, Epidermis/pathology, Epidermis/ultrastructure, Exons, Genes, Recessive, Glucosylceramides/metabolism, Ichthyosis, Lamellar/genetics, Ichthyosis, Lamellar/pathology, Ichthyosis, Lamellar/therapy, Kallikreins/metabolism, Keratinocytes/metabolism, Mice, Models, Biological, Mutation, Permeability, Phenotype, Sequence Analysis, DNA, Skin/metabolism, Skin/pathology, Skin/ultrastructure, Skin Transplantation
Pubmed
Web of science
Open Access
Yes
Create date
16/09/2016 15:32
Last modification date
20/08/2019 17:10
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