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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
This content is Free Access.
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
•
05:16 min
•
May 07, 2020
•
Joel Z. Nordin*
1
,
Yoshitaka Mizobe*
1
,
Harumasa Nakamura
,
Hirofumi Komaki
,
Shin’ichi Takeda
,
Yoshitsugu Aoki
1
Department of Molecular Therapy, National Institute of Neuroscience
,
National Center of Neurology and Psychiatry
,
2
Clinical Research Support Office, Translational Medical Center
,
National Center of Neurology and Psychiatry
,
3
Department of Child Neurology, National Center Hospital
,
National Center of Neurology and Psychiatry
Chapters
00:04
Introduction
01:05
Muscle Sample Preparation
02:10
Muscle Sectioning
03:00
Results: Representative Exon Skipping Efficiency and Dystrophin Quantification
04:23
Conclusion
Summary
Automatic Translation
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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 Skipping
DMD
Antisense Oligonucleotides
Clinical Trials
Sample Preparation
Cryostat Sectioning
RT-PCR
Western Blot
Immunohistochemistry
Hematoxylin And Eosin Staining
Microchip Electrophoresis
Sequencing
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