Investigational Treatments for Epidermolysis Bullosa

Ping Chen Hou, Han Tang Wang, Stasha Abhee, Wei Ting Tu, John A. McGrath, Chao Kai Hsu

研究成果: Review article同行評審

1 引文 斯高帕斯(Scopus)

摘要

Epidermolysis bullosa (EB) is a heterogeneous group of rare inherited blistering skin disorders characterized by skin fragility following minor trauma, usually present since birth. EB can be categorized into four classical subtypes, EB simplex, junctional EB, dystrophic EB and Kindler EB, distinguished on clinical features, plane of blister formation in the skin, and molecular pathology. Treatment for EB is mostly supportive, focusing on wound care and patient symptoms such as itch or pain. However, therapeutic advances have also been made in targeting the primary genetic abnormalities as well as the secondary inflammatory footprint of EB. Pre-clinical or clinical testing of gene therapies (gene replacement, gene editing, RNA-based therapy, natural gene therapy), cell-based therapies (fibroblasts, bone marrow transplantation, mesenchymal stromal cells, induced pluripotential stem cells), recombinant protein therapies, and small molecule and drug repurposing approaches, have generated new hope for better patient care. In this article, we review advances in translational research that are impacting on the quality of life for people living with different forms of EB and which offer hope for improved clinical management.

原文English
頁(從 - 到)801-817
頁數17
期刊American Journal of Clinical Dermatology
22
發行號6
DOIs
出版狀態Published - 2021 十一月

All Science Journal Classification (ASJC) codes

  • 皮膚科

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