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陷的尝试。然而，它被认为是更困难的恢复结构蛋白质比恢复分泌酶。本研究的目的是阐明骨髓移植（BMT）治疗是否可以挽救由角质形成细胞结构蛋白缺陷引起的大疱性表皮病（EB）。BMT治疗成年胶原XVII（Col17）基因敲除小鼠诱导供体来源的角质形成细胞，Col17表达与半桥粒结构的恢复和更好的皮肤表现有关，以及提高存活率。造血干细胞和间充质干细胞在BMT治疗模型中都有产生Col17的潜力。目前常规的BMT技术作为治疗人EB的系统性治疗方法具有显著的潜力。

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.