Surgical Treatment of Wounds Using Stem Cells in Epidermolysis Bullosa (EB)

M. Nita, J. Pliszczyński, A. Eljaszewicz, M. Moniuszko, T. Oldak, K. Woźniak, S. Majewski, C. Kowalewski, A. Kamiński, D. Šladowski, Z. Zimek, M. Kosieradzki, P. Fiedor
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引用次数: 4

Abstract

Epidermolysis bullosa (EB) is a group of hereditary skin diseases, or genodermatoses, characterized by the formation of severe, chronic blisters with painful and life-threatening complications. Despite the previous and ongoing progress in the field, there are still no effective causative treatments for EB. The treatment is limited to relieving symptoms, which—depending on disease severity—may involve skin (blisters, poorly healing wounds caused by the slightest mechanical stimuli, contractures, scarring, pseudosyndactyly) and internal organ abnormalities (esophageal, pyloric, or duodenal atresia; renal failure; and hematopoietic abnormalities). The last decade saw a series of important discoveries that paved the way for new treatment methods, including gene therapy, bone marrow transplantation, cell therapy (allogenic fibroblasts, mesenchymal stem cells [MSCs], and clinical use of induced pluripotent stem cells. Tissue engineering experts are attempting to develop skin-like structures that can facilitate the process of healing to promote skin reconstruction in injuries that are currently incurable. However, this is incredibly challenging, due to the complex structure and the many functions of the skin. Below, we characterize EB and present its potential treatment methods. Despite the cure for EB being still out of reach, recent data from animal models and initial clinical trials in humans have raised patients’, clinicians’, and researchers’ expectations. Consequently, modifying the course of the disease and improving the quality of life have become possible. Moreover, the conclusions drawn based on EB treatment may considerably improve the treatment of other genetic diseases.
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大疱性表皮松解症(EB)干细胞的外科治疗
大疱性表皮松解症(EB)是一组遗传性皮肤病或遗传性皮肤病,其特征是形成严重的慢性水疱,伴有疼痛和危及生命的并发症。尽管该领域过去和现在都取得了进展,但仍然没有有效的EB病因治疗方法。治疗仅限于缓解症状,根据疾病的严重程度,症状可能涉及皮肤(水疱,由最轻微的机械刺激引起的愈合不良的伤口,挛缩,瘢痕形成,假性并指)和内脏器官异常(食管,幽门或十二指肠闭锁;肾功能衰竭;以及造血异常)。在过去的十年中,一系列重要的发现为新的治疗方法铺平了道路,包括基因治疗、骨髓移植、细胞治疗(同种异体成纤维细胞、间充质干细胞[MSCs])和诱导多能干细胞的临床应用。组织工程专家正试图开发类似皮肤的结构,以促进愈合过程,促进目前无法治愈的伤口的皮肤重建。然而,由于皮肤的复杂结构和许多功能,这是非常具有挑战性的。下面,我们将描述EB的特征并介绍其潜在的治疗方法。尽管EB的治愈仍然遥不可及,但最近来自动物模型和人类初步临床试验的数据提高了患者、临床医生和研究人员的期望。因此,改变病程和改善生活质量已成为可能。此外,基于EB治疗得出的结论可能会大大改善其他遗传疾病的治疗。
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