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Duchenne型肌营养不良症(Duchenne muscular dystrophy,DMD)表现为进行性肌肉萎缩,是一种致死性、遗传性神经肌肉疾病。尽管对肌肉萎缩的分子机理的研究取得了很大进展,但是仍然不能治愈,细胞治疗是很有前景的一种治疗方法。成肌细胞移植面临的主要限制是注射后细胞分布差,免疫排斥和细胞存活率低;骨髓干细胞移植需要解决横向分化的低效率问题;而肌源性干细胞看来能更有效地再生表达dystrophin的肌纤维。
Duchenne muscular dystrophy (DMD) manifests itself as progressive muscle atrophy and is a lethal, hereditary neuromuscular disease. Although great progress has been made in the study of the molecular mechanism of muscle wasting, it remains incurable and cell therapy is a promising treatment. The major limitation facing myoblast transplantation is poor cell distribution after injection, low immune rejection and poor cell survival; bone marrow stem cell transplantation needs to address the low efficiency of lateral differentiation; and myogenic stem cells appear to regenerate dystrophin more efficiently Muscle fiber.