Waiting
Login processing...

Trial ends in Request Full Access Tell Your Colleague About Jove
Journal / Medicine / Characterizing Exon Skipping Efficiency in DMD…
JoVE Journal
Medicine

This content is Open Access.

JoVE Journal Medicine
Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
The Monoiodoacetate Model of Osteoarthritis Pain in the Mouse
Journal (Medicine)
The Monoiodoacetate Model of Osteoarthritis Pain in the Mouse
Screening for Melanoma Modifiers using a Zebrafish Autochthonous Tumor Model
Journal (Medicine)
Screening for Melanoma Modifiers using a Zebrafish Autochthonous Tumor Model
 

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

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

Chapters

Summary
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 Skipping Efficiency DMD Patient Samples Clinical Trials Antisense Oligonucleotides Muscle Tissue Preparation Standardized Methodology Reliable Results DMD Field Primers Splicing Therapies RNA And Protein Analysis Tragacanth Gum Cork Discs Isopentane Freezing Specimens
Read Article

Get cutting-edge science videos from JoVE sent straight to your inbox every month.

Waiting X
Simple Hit Counter