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Journal / Medicine / 反义寡核苷酸临床试验中DMD患者样本的Exon跳过效率特征…
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Medicine

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JoVE Journal Medicine
反义寡核苷酸临床试验中DMD患者样本的Exon跳过效率特征
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反义寡核苷酸临床试验中DMD患者样本的Exon跳过效率特征

Article DOI: 10.3791/60672-v • 05:16 min • May 7th, 2020
May 7th, 2020 •
*1, *1, 2, 3, 1, 1
1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 2Clinical Research Support Office, Translational Medical Center, National Center of Neurology and Psychiatry, 3Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry
* These authors contributed equally

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在这里，我们介绍在临床试验中使用桑格测序、RT-PCR和西印迹的肌营养不良素38表达的分子特征。

Tags

医学， 问题 159， 医学， 杜氏肌营养不良症 （DMD）， 临床试验， 肌肉活检， 第一在人类试验， 磷酰胺形态非聚体 （形态或 Pmos）， Dystrophin， 骨骼肌肉， mRNA 拼接， RT - PCR， 西印迹， NS-065/NCNP-01， exon 53 跳过
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