金紫荷,王 荣

杜氏肌营养不良症是一种导致进行性肌肉丢失和过早死亡的毁灭性疾病。虽然医疗管理主要集中在对症治疗，但数十年的研究已经产生了能够恢复受影响的抗肌萎缩蛋白转录本的阅读框或从载体诱导合成截短的抗肌萎缩蛋白蛋白的疗法，以及在临床前或临床发展中基于基因治疗和细胞信号传导的其他策略。本文重点介绍了目前上市的外显子跳跃及基因治疗药物，以期对DMD患者的治疗提供帮助。

杜氏肌营养不良症；外显子跳跃；基因治疗

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