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Charakterisierung der Exon-Skipping-Effizienz in DMD-Patientenproben in klinischen Studien mit Antisense-Oligonukleotiden
JoVE Journal
Medicine
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JoVE Journal Medicine
Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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Charakterisierung der Exon-Skipping-Effizienz in DMD-Patientenproben in klinischen Studien mit Antisense-Oligonukleotiden

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6,236 Views

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05:16 min

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May 07, 2020

DOI:

10.3791/60672-v

DOI:
10.3791/60672-v

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05:16 min
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

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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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