张鹏程;宋亚锋;

• 1:北京体育大学运动人体科学学院
• 2:北京体育大学中国运动与健康研究院

摘要(Abstract)：

杜氏肌营养不良症(DMD)是由肌营养不良蛋白缺失或减少引起的全身肌肉渐进性损伤和运动功能减退的致死性基因遗传病。目前关于DMD患者的治疗手段越来越多,包括药物治疗、物理治疗、基因治疗、干细胞疗法和运动训练疗法等。本文梳理了近20年来载体介导DMD基因治疗情况,发现使用短序列肌营养不良蛋白基因在治疗DMD模型小鼠和犬类身上具有一定效果,并得到一些有效的实验数据证明载体介导基因治疗可以改善动物肌肉形态和功能。但人体临床试验还需要进一步考察载体介导DMD基因治疗的安全性和有效性。

关键词(KeyWords)： 杜氏型肌营养不良症;载体介导基因治疗

Abstract:

Keywords:

基金项目(Foundation):

作者(Author): 张鹏程;宋亚锋;

Email:

DOI: 10.14053/j.cnki.ppcr.202101018

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