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Neurology：丙戊酸有望成为眼咽肌营养不良的治疗手段

2018-07-15 zhangfan MedSci原创

眼咽肌营养不良丙戊酸OPMD
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研究认为，丙戊酸在眼咽肌营养不良模型中表现出良好的肌肉细胞保护作用，有望成为治疗该疾病的潜在方法

近日研究人员考察了丙戊酸 (VPA)对眼咽肌营养不良(OPMD)的治疗潜力。

研究人员使用活细胞成像和生化方法，在OPMD-C2C12 肌肉细胞模型评估VPA潜在的保护作用，该模型致病基因在来自OPMD患者的淋巴母细胞株，通过转基因方法在线虫中表达人突变PABPN1基因。

研究发现，VPA对PABPN1突变体肌肉细胞毒性具有有拮抗作用，可通过增加组蛋白乙酰化水平，调节致病基因移植后细胞生存、增殖和分化。此外，VPA可提高OPMD患者淋巴母细胞株组蛋白乙酰化水平。使用中等浓度VPA处理后，PABPN-13线虫的运动能力得到显著提高。

研究认为，丙戊酸在眼咽肌营养不良模型中表现出良好的肌肉细胞保护作用，有望成为治疗该疾病的潜在方法。

原始出处：


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    2018-07-20 751943081_32627960

    #肌营养不良#

    0

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    2019-02-01 yinhl1978

    #Neurol#

    0

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    2018-07-17 steven_u5

    #丙戊酸#

    0

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    2018-07-15 1ddf0692m34(暂无匿称)

    学习了.长知识

    0

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    2018-07-15 1e0f8808m18(暂无匿称)

    学习了.谢谢分享!

    0