Development of a protein replacement therapy for peeling skin syndrome

Abstract

British Journal of DermatologyVolume 184, Issue 6 p. e197-e197 Plain Language SummaryFree Access Development of a protein replacement therapy for peeling skin syndrome First published: 06 June 2021 https://doi.org/10.1111/bjd.20101AboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinked InRedditWechat Abstract Linked article: Valentin et al. Br J Dermatol 2021; 184:1123–1131. Peeling skin syndrome type 1 is a severe form of congenital ichthyosis. Patients have severe itch and generalized peeling of the skin. Current therapeutic approaches are very unsatisfactory. The disease is the result of mutations (mistakes) in the CDSN gene, which cause a lack of the protein corneodesmosin (CDSN) in the skin. We wanted to develop the first steps towards a specific protein replacement therapy for CDSN deficiency. The aim is to improve the cell–cell connection in two layers of the epidermis (the granular and corneal layers). Human CDSN was produced in bacterial cells. A liposomal carrier system was developed to transport the CDSN to the outer membrane of the epidermal cells. The liposomal carrier system was checked for stability and toxicity. Also, the effect on epidermal cells and artificial skin models was investigated. The liposomes were found to be successfully present at the epidermal cell membranes. The CDSN-deficient skin models were treated with liposomal CDSN and demonstrated the presence of the replaced protein within the granular layer. Finally, the skin barrier test and microscopy showed improved skin integrity (skin that functioned better), if the skin models were treated with liposomal CDSN. We believe that this study presents helpful laboratory results to develop a protein replacement therapy for patients with peeling skin syndrome and other diseases where there is also a lack of CDSN. Volume184, Issue6June 2021Pages e197-e197 RelatedInformation