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

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

DOI:
10.3791/60672-v

•

05:16 min

•

May 07, 2020

•

, , , , ,
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

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

Tags

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

Article

Embed

ADD TO PLAYLIST

Usage Statistics

Related Videos

Slow-release Drug Delivery through Elvax 40W to the Rat Retina: Implications for the Treatment of Chronic Conditions

miRNA Expression Analyses in Prostate Cancer Clinical Tissues

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

Unbiased Deep Sequencing of RNA Viruses from Clinical Samples

Sampling Strategies and Processing of Biobank Tissue Samples from Porcine Biomedical Models

Collecting Hair Samples for Hair Cortisol Analysis in African Americans

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Quantitative [18F]-Naf-PET-MRI Analysis for the Evaluation of Dynamic Bone Turnover in a Patient with Facetogenic Low Back Pain

Inducing and Characterizing Vesicular Steatosis in Differentiated HepaRG Cells

Using a Chemical Biopsy for Graft Quality Assessment

Read Article