Fan Y, Maley M, Beilharz M, Grounds M
Department of Pathology, University of Western Australia, Nedlands, Australia.
Muscle Nerve. 1996 Jul;19(7):853-60. doi: 10.1002/(SICI)1097-4598(199607)19:7<853::AID-MUS7>3.0.CO;2-8.
Myoblast transplantation has been proposed as a potential therapy for Duchenne muscular dystrophy (DMD). A Y-chromosome-specific probe was used to track the fate of donor male myoblasts injected into dystrophic muscles of female mdx mice (which are an animal model for DMD). In situ analysis with the Y-probe showed extremely poor survival of isolated normal male (C57B1/10Sn) donor myoblasts after injection into injured or uninjured muscles of dystrophic (mdx) and normal (C57B1/10Sn) female host mice. A decrease in the numbers of donor (male) myoblasts was seen from 2 days and was marked by 7 days after injection: few or no donor myoblasts were detected in host muscles examined at 3-12 months. There was limited movement of the injected donor myoblasts and fusion into host myofibers was rare. The results of this study strongly suggest that the failure of clinical trials of myoblast transplantation in boys with DMD may have been due to rapid and massive death of the donor myoblasts soon after myoblast injection.
成肌细胞移植已被提议作为杜氏肌营养不良症（DMD）的一种潜在治疗方法。使用Y染色体特异性探针来追踪注入雌性mdx小鼠（DMD的动物模型）营养不良肌肉中的供体雄性成肌细胞的命运。用Y探针进行的原位分析显示，将分离的正常雄性（C57B1/10Sn）供体成肌细胞注入营养不良（mdx）和正常（C57B1/10Sn）雌性宿主小鼠的受伤或未受伤肌肉后，其存活率极低。从注射后2天开始可见供体（雄性）成肌细胞数量减少，到注射后7天明显减少：在3至12个月检查的宿主肌肉中几乎检测不到或未检测到供体成肌细胞。注入的供体成肌细胞移动有限，与宿主肌纤维融合的情况很少见。这项研究的结果强烈表明，DMD男孩成肌细胞移植临床试验失败可能是由于成肌细胞注射后供体成肌细胞迅速大量死亡。
