Mechanism of allogenic bone marrow therapy in epidermolysis bullosa model mice

点击翻译按钮获取中文摘要

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.