Therapeutic strategy for muscular dystrophy by restoring the function of dystroglycan

我们培育了过表达糖基转移酶的转基因小鼠。小鼠出生后生长正常，骨骼肌层粘连蛋白结合活性显著增强。我们将转基因小鼠与先天性肌营养不良的小鼠模型dy^&lt；2J&gt；进行杂交，以检验大量过度表达的治疗效果。然而，高表达的dy^&lt；2J&gt；小鼠的营养不良改变并未得到改善，而肌纤维的变性程度却加重。

We generated transgenic mice overexpressing a glycosyltransferase, LARGE. The mice were born and grew normally, and laminin binding activity of dystroglycan was greatly increased in the skeletal muscle. We crossed the transgenic mice with dy^<2J> mice, a mouse model of congenital muscular dystrophy, to examin therapeutic effect of LARGE overexpression. The dystrophic change of dy^<2J> mice overexpressing LARGE, however, was not improved and the degeneration of myofibers was rather deteriorated.