Su Xuan, Shen Yan, Jin Yue, Jiang Meng, Weintraub Neal, Tang Yaoliang
Vascular Biology Center, Medical College of Georgia, Augusta University; Renji Hospital, School of Medicine, Shanghai Jiaotong University.
Vascular Biology Center, Medical College of Georgia, Augusta University.
J Vis Exp. 2019 Apr 10(146). doi: 10.3791/59320.
Duchene Muscular Dystrophy (DMD) is an X-linked recessive genetic disease caused by a lack of functional dystrophin protein. The disease cannot be cured, and as the disease progresses, the patient develops symptoms of dilated cardiomyopathy, arrhythmia, and congestive heart failure. The DMD mutant mice do not express dystrophin, and are commonly used as a mouse model of DMD. In our recent study, we observed that intramyocardial injection of wide type (WT)-myogenic progenitor cells-derived exosomes (MPC-Exo) transiently restored the expression of dystrophin in the myocardium of DMD mutant mice, which was associated with a transient improvement in cardiac function suggesting that WT-MPC-Exo may provide an option to relieve the cardiac symptoms of DMD. This article describes the technique of MPC-Exo purification and transplantation into hearts of DMD mutant mice.
杜氏肌营养不良症(DMD)是一种由功能性肌营养不良蛋白缺乏引起的X连锁隐性遗传病。该疾病无法治愈,随着病情进展,患者会出现扩张型心肌病、心律失常和充血性心力衰竭等症状。DMD突变小鼠不表达肌营养不良蛋白,常被用作DMD的小鼠模型。在我们最近的研究中,我们观察到向心肌内注射野生型(WT)成肌祖细胞衍生的外泌体(MPC-Exo)可使DMD突变小鼠心肌中肌营养不良蛋白的表达短暂恢复,这与心脏功能的短暂改善有关,表明WT-MPC-Exo可能为缓解DMD的心脏症状提供一种选择。本文介绍了MPC-Exo的纯化技术以及将其移植到DMD突变小鼠心脏中的方法。