日本北海道大学的研究人员日前报告说，他们在利用老鼠做实验时发现，骨髓移植对于表皮松解症有显着疗效。
表皮松解症有多种类型，基本症状是患者皮肤等表皮组织变脆弱，即使是遇到比较轻微的刺激，患者皮肤和黏膜也会出现水疱和糜烂，这种疾病较难治疗。
北海道大学讲师阿部理一领导的研究小组在新一期美国《国家科学院学报》（PNAS）上报告说，他们注意到骨髓中的造血干细胞能发育成皮肤细胞。科研人员人工培育出患有表皮松解症的老鼠，然后为其移植正常老鼠的骨髓。
结果发现，接受骨髓移植的20只患病老鼠的皮肤症状得到改善，它们移植后200天的生存率达到73.7％，而未接受移植的17只老鼠，同一时期的生存率只有27.5％。
据参与研究者介绍，此前表皮松解症只能以对症治疗为主，上述新发现为根治该病带来了希望（蓝建中）。
推荐原文出处：
PNAS doi: 10.1073/pnas.1000044107
Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice
Riichiro Abea,1, Daisuke Inokumaa, Mikako Sasakia, Daichi Hoshinaa, Ken Natsugaa, Wataru Nishiea, James R. McMillana, Hideki Nakamuraa, Tadamichi Shimizub, Masashi Akiyamaa, Daisuke Sawamurac, and Hiroshi Shimizua,1
aDepartment of Dermatology, Hokkaido University Graduate School of Medicine, Sapporo 060-8638, Japan;
bDepartment of Dermatology, Toyama University Graduate School of Medicine and Pharmaceutical Sciences, Toyama 930-0194, Japan; and
cDepartment of Dermatology, Hirosaki University Graduate School of Medicine, Hirosaki 036-8562, Japan
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. However, it is considered more difficult to restore structural proteins than to restore secretory enzymes. This study aims 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. Both hematopoietic and mesenchymal stem cells have the potential to produce Col17 in the BMT treatment model. Furthermore, human cord blood CD34+ cells also differentiated into keratinocytes and expressed human skin component proteins in transplanted immunocompromised (NOD/SCID/γc null) mice. The current conventional BMT techniques have significant potential as a systemic therapeutic approach for the treatment of human EB.
