Christina Guttmann-Gruber, Johann W. Bauer, Josefina Piñón Hofbauer
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Hereditary bullous diseases: current and innovative models to study the skin blistering disease epidermolysis bullosa
As the largest organ of the human body, our skin serves as an interface to the environment, as well as a defensive barrier against dangers therein. Its integrity is facilitated by a complex suprastructural network of proteins that tether the epidermis to the underlying dermis. Mutations in single genes that disrupt the function of these proteins lead to severe bullous disorders such as epidermolysis bullosa (EB). This short review focuses on progress in the establishment of different model systems that recapitulate multiple aspects of the pathological phenotype of EB. These models have been used to decipher disease modifying mechanisms and evaluate therapeutic possibilities aimed at reverting the genetic defect or ameliorating disease-associated complications.
期刊介绍:
Drug Discovery Today: Disease Models discusses the non-human experimental models through which inference is drawn regarding the molecular aetiology and pathogenesis of human disease. It provides critical analysis and evaluation of which models can genuinely inform the research community about the direct process of human disease, those which may have value in basic toxicology, and those which are simply designed for effective expression and raw characterisation.