Duchenne型肌营养不良症的治疗
收稿日期: 2020-11-11
网络出版日期: 2021-02-05
基金资助
国家自然科学基金——DYS△45-55基因修复的人脂肪干细胞源性肌肉祖细胞移植DMD模型鼠促进肌肉再生的研究(81801246);实验性大脑皮层梗死后同侧丘脑Aβ沉积分布特征及其机制研究(81171084);广东省自然科学基金——Dystrophin缺陷基因修复的脂肪干细胞源性肌肉祖细胞移植DMD模型鼠促进肌肉再生及成肌机制的研究(2018A030313636);经典途径细胞焦亡在实验性大脑皮层梗死后同侧丘脑继发性损害中的作用及其机制研究(2020A1515011249);广东省科技计划项目——正常人来源的脂肪干细胞心内注射移植mdx鼠对心脏结构和功能的作用及其机制的研究(2017A020215094)
Duchenne型肌营养不良症(Duchenne muscular dystrophy, DMD)是一种X连锁致死性遗传性肌病,由Dystrophin基因突变导致抗肌萎缩蛋白缺失所致。DMD尚缺乏有效的治疗方法,但随着对该病发病机制和病理变化过程的认识不断深入,其治疗选择越来越多。这些治疗方法旨在恢复Dystrophin蛋白表达或弥补Dystrophin蛋白缺失。
关键词： 肌营养不良; 杜氏; dystrophin蛋白; 基因治疗; 外显子跳跃
罗宏 , 岑海媚 , 罗彬 , 张玉生 , 张誉 . Duchenne型肌营养不良症的治疗[J]. 临床荟萃, 2021 , 36(2) : 168 -173 . DOI: 10.3969/j.issn.1004-583X.2021.02.015
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