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Duchenne型肌营养不良症是我国常见的X连锁隐性遗传性肌病。目前广泛应用的动物模型是mdx小鼠,但其没有很好地模拟人类疾病特点。最近,Sacco等报导了一个新的小鼠模型mdx/mTRG2,它不仅有抗肌萎缩蛋白的缺陷,还有端粒酶的缺失,较好地模拟了人类疾病的症状。通过该模型,人们认识到抗肌萎缩蛋白的缺陷引起肌细胞退化,肌肉干细胞被激活对抗其退化,但干细胞的过度增殖又导致端粒长度下降,引起肌肉干细胞增殖能力的衰竭,最终产生了肌营养不良的表型。该模型使人们对Duchenne型肌营养不良症的发病机制有了进一步的理解,为其治疗提供了新的研究平台。
Duchenne muscular dystrophy is a common X-linked recessive myopathic myopathy in China. The currently widely used animal model is mdx mice, but it does not well model the characteristics of human disease. Recently, Sacco et al. Reported a new mouse model, mdx / mTRG2, which not only has the defect of dystrophin but also has the deletion of telomerase, which better mimics the symptoms of human diseases. Through this model, it is recognized that defects in dystrophin cause muscle cell degeneration, muscle stem cells are activated against degradation, but excessive proliferation of stem cells in turn leads to telomere length decline, causing failure of muscle stem cell proliferation, resulting in muscle Malnutrition phenotype. The model gives a further understanding of the pathogenesis of Duchenne muscular dystrophy and provides a new platform for its treatment.