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Journal / Medicine / Karakteriseren exon overslaan efficiëntie in DMD…
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Medicine

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JoVE Journal Medicine
Karakteriseren exon overslaan efficiëntie in DMD patiënt monsters in klinische proeven van antisense oligonucleotiden
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Karakteriseren exon overslaan efficiëntie in DMD patiënt monsters in klinische proeven van antisense oligonucleotiden

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 presenteren we de moleculaire karakterisering van dystrofine 38 expressie met behulp van Sanger sequencing, RT-PCR, en westerse blotting in de klinische studie.

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Geneeskunde Geneeskunde Duchenne spierdystrofie (DMD) Klinische studie Spierbiopsie Eerste in menselijke proef Phosphorodiamidate morpholino oligomers (morpholinos of PMOs) Dystrophin Skeletale spier mRNA splicing RT-PCR Western blotting NS-065/NCNP-01 exon 53 skipping
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