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Journal / Medicine / Antisense Oligonükleotidlerin Klinik…
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JoVE Journal Medicine
Antisense Oligonükleotidlerin Klinik Çalışmalarında DMD Hasta Örneklerinde Exon Atlama Etkinliğinin Karakterize Edilmesi
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Antisense Oligonükleotidlerin Klinik Çalışmalarında DMD Hasta Örneklerinde Exon Atlama Etkinliğinin Karakterize Edilmesi

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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Burada klinik çalışmada Sanger sıralama, RT-PCR ve batı lekeleme kullanarak distrofin 38 ekspresyonunun moleküler karakterizasyonunu satMaktadır.

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Tıp Sayı 159 Tıp Duchenne musküler distrofi (DMD) Klinik çalışma Kas Biyopsisi İnsan deneyinde ilk Fosforodiamiat morkoliolino oligomerler (morfuz veya PmOs) Distrofin İskelet kası mRNA yapıştırma RT-PCR Batı lekeleme NS-065/NCNP-01 exon 53 atlama
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