赛业DMD人源化小鼠模型，助力“被定时的生命”重获多彩人生

有这样一种疾病，其发病患者通常是3-5岁的孩童，患者一开始会出现进行性肌无力症状，在12岁左右慢慢失去了行走能力，它就是杜氏肌营养不良症（Duchenne muscular dystrophy, DMD）。

近年来，在社会各方力量的持续关注和努力下，国内对DMD的社会认知度有了一定的提高，诊疗技术、药物研发也在不断向前推进着。据悉，Sarepta Therapeutics公司已向FDA提交了一份生物制品许可申请（BLA），SRP-9001有望成为第一款通过AAV治疗DMD的基因疗法；Cure Rare Disease也在今年取得新进展，其研发的CRD-TMH-001是首款获批进入DMD临床试验阶段的CRISPR基因编辑疗法。

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