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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
JoVE 杂志
医学
This content is Free Access.
JoVE 杂志 医学
Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

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

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Here, we present the molecular characterization of dystrophin 38 expression using Sanger sequencing, RT-PCR, and western blotting in the clinical trial.

Tags

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

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