Epidermolysis bullosa (EB) is a group of rare genetic skin fragility disorders, which are hereditary. These disorders are associated with mutations in at least 16 genes that encode components of the epidermal adhesion complex. Currently, there are no effective treatments for this disorder. All current treatment approaches focus on topical treatments to prevent complications and infections. In recent years, significant progress has been achieved in the treatment of the severe genetic skin blistering condition known as EB through preclinical and clinical advancements. Promising developments have emerged in the areas of protein and cell therapies, such as allogeneic stem cell transplantation; in addition, RNA-based therapies and gene therapy approaches have also become a reality. Stem cells obtained from embryonic or adult tissues, including the skin, are undifferentiated cells with the ability to generate, maintain, and replace fully developed cells and tissues. Recent advancements in preclinical and clinical research have significantly enhanced stem cell therapy, presenting a promising treatment option for various diseases that are not effectively addressed by current medical treatments. Different types of stem cells such as primarily hematopoietic and mesenchymal, obtained from the patient or from a donor, have been utilized to treat severe forms of diseases, each with some beneficial effects. In addition, extensive research has shown that gene transfer methods targeting allogeneic and autologous epidermal stem cells to replace or correct the defective gene are promising. These methods can regenerate and restore the adhesion of primary keratinocytes in EB patients. The long-term treatment of skin lesions in a small number of patients has shown promising results through the transplantation of skin grafts produced from gene-corrected autologous epidermal stem cells. This article attempts to summarize the current situation, potential development prospects, and some of the challenges related to the cell therapy approach for EB treatment.

大疱性表皮松解症（EB）是一组罕见的遗传性皮肤脆性疾病。这些疾病与至少 16 个编码表皮粘附复合物成分的基因的突变有关。目前，这种疾病没有有效的治疗方法。目前所有的治疗方法都集中于局部治疗以预防并发症和感染。近年来，通过临床前和临床进展，在严重遗传性皮肤起泡疾病（EB）的治疗方面取得了重大进展。蛋白质和细胞疗法领域出现了有希望的发展，例如同种异体干细胞移植；此外，基于RNA的疗法和基因疗法也已成为现实。从胚胎或成人组织（包括皮肤）获得的干细胞是未分化的细胞，能够生成、维持和替换完全发育的细胞和组织。临床前和临床研究的最新进展显着增强了干细胞疗法，为当前医学治疗无法有效解决的各种疾病提供了有前景的治疗选择。从患者或捐赠者获得的不同类型的干细胞，例如造血干细胞和间充质干细胞，已被用来治疗严重的疾病，每种干细胞都有一些有益的作用。此外，广泛的研究表明，针对同种异体和自体表皮干细胞来替换或纠正缺陷基因的基因转移方法是有前途的。这些方法可以再生并恢复 EB 患者原代角质形成细胞的粘附力。通过移植由基因校正的自体表皮干细胞产生的皮肤移植物，对少数患者的皮肤病变进行长期治疗已显示出有希望的结果。本文试图总结 EB 细胞治疗方法的现状、潜在发展前景以及一些挑战。