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Functional therapies for cutaneous wound repair in epidermolysis bullosa
Advanced Drug Delivery Reviews ( IF 16.1 ) Pub Date : 2017-12-15 , DOI: 10.1016/j.addr.2017.12.003
Patricia Peking , Ulrich Koller , Eva M. Murauer

Chronic wounding as a result of recurrent skin blistering in the painful genetic skin disease epidermolysis bullosa, may lead to life-threatening infections, increased risk of tumor formation, and other serious medical complications. Therefore, epidermolysis bullosa patients have an urgent need for optimal wound care and tissue regeneration. Therapeutic strategies using gene-, protein-, and cell-therapies are being developed to improve clinical symptoms, and some of them have already been investigated in early clinical trials. The most favorable options of functional therapies include gene replacement, gene editing, RNA targeting, and harnessing natural gene therapy. This review describes the current progress of the different approaches targeting autologous skin cells, and will discuss the benefits and challenges of their application.



中文翻译:

大疱表皮松解术中皮肤伤口修复的功能疗法

疼痛的遗传性皮肤病大疱性表皮松解症中反复出现皮肤水疱引起的慢性伤口,可能导致威胁生命的感染,增加肿瘤形成的风险以及其他严重的医疗并发症。因此,大疱性表皮松解症患者迫切需要最佳的伤口护理和组织再生。正在开发使用基因,蛋白质和细胞疗法的治疗策略,以改善临床症状,其中一些已经在早期临床试验中进行了研究。功能疗法的最有利选择包括基因替换,基因编辑,RNA靶向和利用天然基因疗法。这篇综述描述了针对自体皮肤细胞的不同方法的最新进展,并将讨论其应用的好处和挑战。

更新日期:2017-12-15
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