Mechanism of allogenic bone marrow therapy in epidermolysis bullosa model mice

根据干细胞可塑性的概念，已经报道了使用骨髓来源的细胞治疗先天性蛋白质缺乏症的尝试。然而，恢复结构蛋白被认为比恢复分泌酶更难。本研究旨在探讨骨髓移植(BMT)能否挽救角质形成细胞结构蛋白缺陷引起的大疱性表皮松解症(EB)。BMT治疗成年XVII(Col17)基因敲除小鼠可诱导供体来源的角质形成细胞，Col17的表达与半染色体结构的恢复和皮肤表现的改善有关，并提高了存活率。在骨髓移植治疗模型中，造血干细胞和间充质干细胞都有产生Col17的潜力。目前常规的骨髓移植技术作为治疗人类EB的一种系统治疗方法具有很大的潜力。

Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported according to the concepts of stem cell plasticity. However, it is considered more difficult to restore structural proteins than to restore secretary enzymes. The aim of the study is to clarify whether bone marrow transplantation (BMT) treatment can rescue epidermolysis bullosa (EB) caused by the defect of keratinocyte structural proteins. BMT treatment of adult collagen XVII (Col17) knockout mice induced donor-derived keratinocytes, Col17 expression associated with the recovery of hemidesmosomal structure and better skin manifestations, as well improving the survival rate. Both hematopoietic and mesenchymal stem cells have the potential to produce Col17 in the BMT treatment model. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB.