重组腺相关病毒载体介导的迷你肌萎缩蛋白基因SMCKA3999改善mdx小鼠肌电图和营养不良肌肉收缩功能

Q4 Medicine

中华神经科杂志 Pub Date : 2005-09-01 DOI:10.3760/J.ISSN:1006-7876.2005.09.007

H. Li, Su-ming Zhang, Chun-lian Chen, Xiao Qin Hu, Si-yu Fang, Xiao Xiao, Daowen Wang

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引用次数: 0

摘要

目的研究重组腺相关病毒载体(rAAV)介导的人抗肌萎缩蛋白(dystrophin)小基因SMCKA3999对Duchenne肌营养不良(DMD)在病理、肌电图、肌力方面的治疗作用. 方法将dystrophin小基因SMCKA3999克隆至rAAV并包装成rAAV SMCKA3999病毒,以5×109病毒颗粒多点注射于DMD模型鼠(mdx鼠)腓肠肌,基因治疗4个月后,以免疫荧光双标法检测肌膜dystrophin基因表达,采用Nicolet肌电及诱发电位仪记录mdx鼠肌电图,基因治疗5个月后以肌肉离体灌注电刺激法测定腓肠肌肌力,观察rAAV SMCKA3999对DMD动物模型鼠的治疗作用. 结果 rAAV SMCKA3999能有效稳定表达,并使肌膜缺失的dystrophin恢复,明显改善DMD肌电图表现,肌力恢复. 结论 rAAV SMCKA3999对mdx鼠治疗有效,能显著改善其肌肉功能,应用rAAV介导的dystrophin小基因SMCKA3999基因治疗是临床治疗DMD有希望的方法。

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Recombinant adeno-associated virus vector-mediated minidystrophin gene SMCKA3999 improves EMG and dystrophic muscle contractile function in mdx mice

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