Dystrophin expression in the mdx mouse restored by stem cell transplantation
Dystrophin expression in the mdx mouse restored by stem cell transplantation
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DOI：
10.1038/43919
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发表时间：
1999-09-23
期刊：
影响因子：
64.8
通讯作者：
Mulligan, RC
中科院分区：
文献类型：
--
作者：
Gussoni, E;Soneoka, Y;Mulligan, RC
由于无法将细胞或基因分散递送至受影响的组织或器官，细胞或基因疗法对于涉及广泛分布于全身的细胞的疾病的开发受到严重阻碍(1)。在此，我们报告了 mdx 小鼠（一种杜兴氏肌营养不良症动物模型）的骨髓移植研究结果(2)，该结果表明，将正常造血干细胞或新型肌肉源性干细胞静脉注射到受辐射的动物体内，可导致移植受体的造血室重建、供体来源的细胞核掺入肌肉以及受影响的肌营养不良蛋白表达部分恢复肌肉。这些结果表明，使用骨髓移植程序移植不同的干细胞群可能为治疗肌营养不良症以及其他疾病提供意想不到的途径，在这些疾病中，将治疗细胞全身输送到全身各处至关重要。我们的研究还表明，从不同组织或器官中分离出的干细胞的固有发育潜力可能比之前预期的更加相似。
The development of cell or gene therapies for diseases involving cells that are widely distributed throughout the body has been severely hampered by the inability to achieve the disseminated delivery of cells or genes to the affected tissues or organ(1). Here we report the results of bone marrow transplantation studies in the mdx mouse, an animal model of Duchenne's muscular dystrophy(2), which indicate that the intravenous injection of either normal haematopoietic stem cells or a novel population of muscle-derived stem cells into irradiated animals results in the reconstitution of the haematopoietic compartment of the transplanted recipients, the incorporation of donor-derived nuclei into muscle, and the partial restoration of dystrophin expression in the affected muscle. These results suggest that the transplantation of different stem cell populations, using the procedures of bone marrow transplantation, might provide an unanticipated avenue for treating muscular dystrophy as well as other diseases where the systemic delivery of therapeutic cells to sites throughout the body is critical. Our studies also suggest that the inherent developmental potential of stem cells isolated from diverse tissues or organs may be more similar than previously anticipated.