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Journal / Medicine / Caratterizzare l'efficienza di salto di esone nei …
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Caratterizzare l'efficienza di salto di esone nei campioni di pazienti DMD negli studi clinici sugli oligonucleotidi antisenso
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Caratterizzare l'efficienza di salto di esone nei campioni di pazienti DMD negli studi clinici sugli oligonucleotidi antisenso

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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Qui, presentiamo la caratterizzazione molecolare dell'espressione distrofina 38 usando il sequenziamento Sanger, RT-PCR e il gonfiore occidentale nello studio clinico.

Tags

Medicina Problema 159 Medicina Distrofia muscolare di Duchenne (DMD) Studio clinico Biopsia muscolare Primo nella sperimentazione umana oligomeri morpholino di fosforodiamidati (morpholinos o PMO) Distrofina Muscolo scheletrico splicing mRNA RT-PCR gonfiore occidentale NS-065/NCN
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