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大疱性表皮松解症的认识与治疗新进展

Recent advances in understanding and managing epidermolysis bullosa.

作者信息

Kiritsi Dimitra, Nyström Alexander

机构信息

Department of Dermatology, Medical Center-University of Freiburg, Faculty of Medicine, 79104 Freiburg, Germany.

出版信息

F1000Res. 2018 Jul 17;7. doi: 10.12688/f1000research.14974.1. eCollection 2018.

Abstract

Epidermolysis bullosa (EB) is a clinically and genetically heterogeneous skin fragility disorder characterized by trauma-induced skin dissociation and the development of painful wounds. So far, mutations in 20 genes have been described as being associated with more than 30 clinical EB subtypes. The era of whole-exome sequencing has revolutionized EB diagnostics with gene panels being developed in several EB centers and allowing quicker diagnosis and prognostication. With the advances of gene editing, more focus has been placed on gene editing-based therapies for targeted treatment. However, their implementation in daily care will still take time. Thus, a significant focus is currently being placed on achieving a better understanding of the pathogenetic mechanisms of each subtype and using this knowledge for the design of symptom-relief therapies, i.e. treatment options aimed at ameliorating and not curing the disease.

摘要

大疱性表皮松解症(EB)是一种临床和遗传异质性的皮肤脆性疾病,其特征为创伤诱导的皮肤分离和疼痛性伤口的形成。迄今为止,已描述了20个基因中的突变与30多种临床EB亚型相关。全外显子测序时代彻底改变了EB的诊断,多个EB中心已开发出基因检测板,能够实现更快的诊断和预后评估。随着基因编辑技术的进步,更多的重点放在了基于基因编辑的靶向治疗上。然而,将这些技术应用于日常护理仍需时日。因此,目前的一个重要重点是更好地理解每种亚型的发病机制,并利用这些知识设计症状缓解疗法,即旨在改善而非治愈该疾病的治疗方案。

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