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Journal / Medicine / Karaktärisera Exon Hoppa effektivitet i DMD…
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Medicine

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JoVE Journal Medicine
Karaktärisera Exon Hoppa effektivitet i DMD patientprover i kliniska prövningar av Antisense Oligonukleotider
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Karaktärisera Exon Hoppa effektivitet i DMD patientprover i kliniska prövningar av Antisense Oligonukleotider

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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Här presenterar vi den molekylära karakterisering av dystrofin 38 uttryck med sanger sekvensering, RT-PCR och västra blotting i den kliniska prövningen.

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Medicin Medicin Duchennes muskeldystrofi (DMD) Klinisk prövning Muskelbiopsi Först i mänsklig prövning Fosfordiamidat morpholino oligomerer (morfolinos eller PMOs) Dystrophin Skelettmuskel mRNA splitsar RT-PCR Western blotting NS-065/NCNP-01 exon 53 hoppa över
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