Abe Riichiro
Department of Dermatology, Hokkaido University Graduate School of Medicine.
Nihon Rinsho Meneki Gakkai Kaishi. 2011;34(2):85-90. doi: 10.2177/jsci.34.85.
Attempts to treat congenital protein deficiencies using bone marrow-derived cells have been reported. These efforts have been based on the concepts of stem cell plasticity. We aimed to clarify whether bone marrow transplantation (BMT) treatment can rescue epidermolysis bullosa (EB) caused by defects in keratinocyte structural proteins. BMT treatment of adult collagen XVII (Col17) knockout mice induced donor-derived keratinocytes and Col17 expression associated with the recovery of hemidesmosomal structure and better skin manifestations, as well improving the survival rate. Furthermore, human cord blood CD34+ cells also differentiated into keratinocytes and expressed human skin component proteins in transplanted immunocompromised mice. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB.
已有报道尝试使用骨髓来源的细胞治疗先天性蛋白质缺乏症。这些努力基于干细胞可塑性的概念。我们旨在阐明骨髓移植（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松解症（EB）。对成年胶原 XVII（Col17）基因敲除小鼠进行BMT治疗，可诱导供体来源的角质形成细胞和Col17表达，这与半桥粒结构的恢复和更好的皮肤表现相关，同时也提高了存活率。此外，人脐带血CD34+细胞在移植的免疫缺陷小鼠中也分化为角质形成细胞并表达人皮肤组成蛋白。当前的传统BMT技术作为治疗人类EB的全身性治疗方法具有巨大潜力。
