遗传性大疱性疾病:研究皮肤起疱性疾病大疱性表皮松解症的现有和创新模型

Q3 Pharmacology, Toxicology and Pharmaceutics
Christina Guttmann-Gruber, Johann W. Bauer, Josefina Piñón Hofbauer
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引用次数: 4

摘要

作为人体最大的器官,我们的皮肤是与环境接触的界面,也是抵御环境危险的防御屏障。它的完整性是由一个复杂的蛋白质上层结构网络促进的,它将表皮连接到下层的真皮层。破坏这些蛋白功能的单基因突变可导致严重的大疱性疾病,如大疱性表皮松解症(EB)。这篇简短的综述着重于建立不同模型系统的进展,这些模型系统概括了EB病理表型的多个方面。这些模型已被用于破译疾病修饰机制,并评估旨在恢复遗传缺陷或改善疾病相关并发症的治疗可能性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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.

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来源期刊
Drug Discovery Today: Disease Models
Drug Discovery Today: Disease Models Pharmacology, Toxicology and Pharmaceutics-Drug Discovery
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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.
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