Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells
Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells
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DOI：
10.1182/blood-2008-06-161299
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发表时间：
2009-01-29
期刊：
影响因子：
20.3
通讯作者：
Blazar, Bruce R.
中科院分区：
文献类型：
--
作者：
Tolar, Jakub;Ishida-Yamamoto, Akemi;Blazar, Bruce R.
隐性营养不良型大疱性表皮松解症 (RDEB) 是一种由于 VII 型胶原蛋白基因 (Col7a1) 突变引起的无法治愈的皮肤脆弱和水疱疾病。由于缺乏由 VII 型胶原蛋白组成的锚定原纤维，缺乏 VII 型胶原蛋白的产生会导致基底膜区域的粘附力丧失。我们报告说，在由靶向 Col7a1 破坏产生的 RDEB 小鼠模型中，野生型同源骨髓细胞归巢于受损皮肤，产生 VII 型胶原蛋白和锚定原纤维，改善皮肤脆性，并降低致死率。这些数据提供了第一个证据，证明骨髓细胞群可以纠正 RDEB 小鼠中发现的基底膜区缺陷，并为治疗人类 RDEB 和其他细胞外基质疾病提供潜在有价值的方法。 （血。2009；113：1167-1174）
The recessive dystrophic form of epidermolysis bullosa (RDEB) is a disorder of incurable skin fragility and blistering caused by mutations in the type VII collagen gene (Col7a1). The absence of type VII collagen production leads to the loss of adhesion at the basement membrane zone due to the absence of anchoring fibrils, which are composed of type VII collagen. We report that wild-type, congenic bone marrow cells homed to damaged skin, produced type VII collagen protein and anchoring fibrils, ameliorated skin fragility, and reduced lethality in the murine model of RDEB generated by targeted Col7a1 disruption. These data provide the first evidence that a population of marrow cells can correct the basement membrane zone defect found in mice with RDEB and offer a potentially valuable approach for treatment of human RDEB and other extracellular matrix disorders. (Blood. 2009;113:1167-1174)