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Medicine

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多外显子跳跃在犬X连锁肌营养不良用鸡尾酒反义寡核苷酸
 
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多外显子跳跃在犬X连锁肌营养不良用鸡尾酒反义寡核苷酸

Article doi: 10.3791/53776
May 24th, 2016

Summary May 24th, 2016

Please note that all translations are automatically generated.

Click here for the English version.

外显子跳跃目前是杜氏肌营养不良症(DMD)一个最有希望的治疗选择。扩大对DMD患者的适用性和优化所得截短的肌营养不良蛋白的稳定性/功能,一个多外显子用鸡尾酒的反义寡核苷酸跳过方法被开发,我们证实在狗模型全身肌营养不良蛋白救援。

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