Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix
Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix
复制标题
DOI：
10.1016/s0140-6736(13)61897-8
复制
发表时间：
2013-10-05
期刊：
影响因子：
168.9
通讯作者：
Wagner, John E.
中科院分区：
文献类型：
--
作者：
Tolar, Jakub;Wagner, John E.
与过去几十年流行的专业观点相反，最近的实验和临床数据支持这样一个事实，即异体血液和骨髓移植的蛋白质替代疗法不仅限于酶等自由扩散的分子，而且还包括胶原等大结构蛋白。一个典型的例子是广泛性重度隐性营养不良大疱性表皮松解症中VII型胶原缺乏的交叉矫正，其中血液和骨髓移植可以减轻疾病的粘膜皮肤表现，提高患者的生活质量。虽然同种异体血液和骨髓移植可以改善皮肤和粘膜的完整性，但今天的成就只是治愈漫漫长路上的第一步。未来的战略将建立在从这些首次移植研究中吸取教训的基础上。
Contrary to the prevailing professional opinion of the past few decades, recent experimental and clinical data support the fact that protein replacement therapy by allogeneic blood and marrow transplantation is not limited to freely diffusible molecules such as enzymes, but also large structural proteins such as collagens. A prime example is the cross-correction of type VII collagen deficiency in generalised severe recessive dystrophic epidermolysis bullosa, in which blood and marrow transplantation can attenuate the mucocutaneous manifestations of the disease and improve patients' quality of life. Although allogeneic blood and marrow transplantation can improve the integrity of the skin and mucous membranes, today's accomplishments are only the first steps on the long pathway to cure. Future strategies will be built on the lessons learned from these first transplant studies.