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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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May 07, 2020
DOI:
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
概要
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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.
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Nordin, J. Z., Mizobe, Y., Nakamura, H., Komaki, H., Takeda, S., Aoki, Y. Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides. J. Vis. Exp. (159), e60672, doi:10.3791/60672 (2020).
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