Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes

Junctional epidermolysis bullosa caused by loss-of-function variants in genes encoding the skin basement membrane proteins laminin 332, type XVII collagen, or integrin α6β4 affects patients from birth with severe blistering, eventually leading to scarring and early lethality. In this study, we have...

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Main Authors: Eleri Mai Jones, Priya Garcha, Monique Aumailley, Edel Anne O’Toole, Emanuel Rognoni, Matthew Caley
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:JID Innovations
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Online Access:http://www.sciencedirect.com/science/article/pii/S2667026724000791
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author Eleri Mai Jones
Priya Garcha
Monique Aumailley
Edel Anne O’Toole
Emanuel Rognoni
Matthew Caley
author_facet Eleri Mai Jones
Priya Garcha
Monique Aumailley
Edel Anne O’Toole
Emanuel Rognoni
Matthew Caley
author_sort Eleri Mai Jones
collection DOAJ
description Junctional epidermolysis bullosa caused by loss-of-function variants in genes encoding the skin basement membrane proteins laminin 332, type XVII collagen, or integrin α6β4 affects patients from birth with severe blistering, eventually leading to scarring and early lethality. In this study, we have optimized a previously published junctional epidermolysis bullosa–knockout mouse model with weekly tamoxifen intraperitoneal injections, resulting in a more controllable and severe model. Owing to the titratable dosing, this model now recapitulates both early and advanced stages of the human disease, strengthening its use in therapeutic studies. The gradual loss of laminin-α3 in the skin of the mouse through weekly injections lead to generalized blistering and fibrotic dermal changes in multiple skin sites by week 12 after tamoxifen. Our findings demonstrate the usefulness of optimizing tamoxifen induction in Cre-loxP mouse models of extracellular matrix proteins, an approach that could be applicable to other emerging inducible transgenic disease models to improve their ability to mimic the human disease phenotype.
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spelling doaj-art-f8b593b097f14e7ca3efbfd91a9fab5e2025-08-20T02:57:33ZengElsevierJID Innovations2667-02672025-03-015210033110.1016/j.xjidi.2024.100331Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease PhenotypesEleri Mai Jones0Priya Garcha1Monique Aumailley2Edel Anne O’Toole3Emanuel Rognoni4Matthew Caley5Cell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United KingdomCell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United KingdomCentre for Biochemistry, Medical Faculty, University of Cologne, Cologne, GermanyCell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United KingdomCell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United Kingdom; Correspondence: Emanuel Rognoni, Cell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United Kingdom.Cell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United Kingdom; Matthew Caley, Cell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United Kingdom.Junctional epidermolysis bullosa caused by loss-of-function variants in genes encoding the skin basement membrane proteins laminin 332, type XVII collagen, or integrin α6β4 affects patients from birth with severe blistering, eventually leading to scarring and early lethality. In this study, we have optimized a previously published junctional epidermolysis bullosa–knockout mouse model with weekly tamoxifen intraperitoneal injections, resulting in a more controllable and severe model. Owing to the titratable dosing, this model now recapitulates both early and advanced stages of the human disease, strengthening its use in therapeutic studies. The gradual loss of laminin-α3 in the skin of the mouse through weekly injections lead to generalized blistering and fibrotic dermal changes in multiple skin sites by week 12 after tamoxifen. Our findings demonstrate the usefulness of optimizing tamoxifen induction in Cre-loxP mouse models of extracellular matrix proteins, an approach that could be applicable to other emerging inducible transgenic disease models to improve their ability to mimic the human disease phenotype.http://www.sciencedirect.com/science/article/pii/S2667026724000791Inducible transgenic modelJunctional EBLamininSkin inflammation
spellingShingle Eleri Mai Jones
Priya Garcha
Monique Aumailley
Edel Anne O’Toole
Emanuel Rognoni
Matthew Caley
Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes
JID Innovations
Inducible transgenic model
Junctional EB
Laminin
Skin inflammation
title Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes
title_full Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes
title_fullStr Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes
title_full_unstemmed Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes
title_short Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes
title_sort weekly intraperitoneal injection of tamoxifen in an inducible in vivo model of junctional epidermolysis bullosa generates early and advanced disease phenotypes
topic Inducible transgenic model
Junctional EB
Laminin
Skin inflammation
url http://www.sciencedirect.com/science/article/pii/S2667026724000791
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