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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
JoVE Journal
Medicine
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JoVE Journal Medicine
Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
Please note that all translations are automatically generated. Click here for the English version.

反义寡核苷酸临床试验中DMD患者样本的Exon跳过效率特征

DOI:
10.3791/60672-v

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05:16 min

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May 07, 2020

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, , , , ,
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

Chapters

  • 00:04Introduction
  • 01:05Muscle Sample Preparation
  • 02:10Muscle Sectioning
  • 03:00Results: Representative Exon Skipping Efficiency and Dystrophin Quantification
  • 04:23Conclusion

Summary

Automatic Translation

Automatic Translation

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

Exon SkippingDMDAntisense OligonucleotidesClinical TrialsSample PreparationCryostat SectioningRT-PCRWestern BlotImmunohistochemistryHematoxylin And Eosin StainingMicrochip ElectrophoresisSequencing

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