Duchenne肌营养不良模型鼠骨髓移植后dystrophin的表达

Dystrophin expression in mdx mouse restored by bone marrow transplantation

目的 观察Duchenne型肌营养不良模型鼠 (mdx鼠 )不同成分骨髓细胞移植后dystrophin的表达。方法 用体外培养的C57BL雄鼠的骨髓细胞、骨髓悬浮细胞、骨髓基质细胞分别经鼠尾静脉注射到放疗后的mdx鼠体内 ,动态观察受体雌性mdx鼠骨骼肌dystrophin的表达 ,并取受体mdx雌鼠血 ,用聚合酶链反应进行Sry基因检测。结果 骨髓细胞、悬浮细胞、基质细胞移植后 1～ 2个月 ,较少肌纤维表达dystrophin(<1 % ) ,3～ 4个月分别约有 7%、6 %、4 %的肌纤维表达dystrophin。Sry基因检测均扩增出受体雌鼠Y染色体上 449bp的DNA片段 ,提示供体细胞在受体内存活。结论 骨髓细胞、悬浮细胞、基质细胞分别移植到mdx鼠体内后 。

Objective Duchenne muscular dystrophy (DMD) is a lethal inherited disorder; the main pathogenesis is deficiency of dystrophin Our study is to observe dystrophin expression in myofibers of mdx mice (an animal model of DMD) transplanted with different bone marrow cells Methods Bone marrow cells, suspension cells and stromal cells were cultured in vitro from C57BL male mice, and these cells were transplanted respectively by tail vein of irradiated mdx mice Dystrophin expression of female mdx mice was detected dynamically By using PCR technique, Y chromosome specific sex determining regions were detected dynamically with the blood of transplanted female mdx mice Results Few dystrophin expression in myofibers (<1%) could be seen under fluorescent microscope at 1 to 2 months after marrow cell, suspension cell, and stromal cell transplantation At 3 to 4 months, about 7%, 6%, and 4% myofibers expressed dystrophin By using PCR technique, DNA specific fragments of 449 bp were amplified in female mdx mice It showed that the donor's cells survived in the recipients Conclusions This study shows that most mdx mice can survive by being transplanted bone marrow cells, suspension cells, and stromal cells Dystrophin can be expressed by different component of marrow cell transplantation It is possible to provide a new method for DMD treatment.