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Journal / Medicine / Charakterisierung der Exon-Skipping-Effizienz in…
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Medicine

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JoVE Journal Medicine
Charakterisierung der Exon-Skipping-Effizienz in DMD-Patientenproben in klinischen Studien mit Antisense-Oligonukleotiden
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Charakterisierung der Exon-Skipping-Effizienz in DMD-Patientenproben in klinischen Studien mit Antisense-Oligonukleotiden

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

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Hier stellen wir die molekulare Charakterisierung der Dystrophin-38-Expression mit Sanger-Sequenzierung, RT-PCR und Western Blotting in der klinischen Studie vor.

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Medizin Ausgabe 159 Medizin Duchenne-Muskeldystrophie (DMD) Klinische Studie Muskelbiopsie Erste in der Humanstudie Phosphorodiamid-Morpholino-Oligomere (Morpholinos oder PMOs) Dystrophin Skelettmuskel mRNA-Spleißen RT-PCR Western Blotting NS-065/NCNP-01 exon 53 skipping
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