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Caracterizando Exon Pulando Eficiência em Amostras de Pacientes DMD em Ensaios Clínicos de Oligonucleotídeos Antisamescenos
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Caracterizando Exon Pulando Eficiência em Amostras de Pacientes DMD em Ensaios Clínicos de Oligonucleotídeos Antisamescenos

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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Aqui, apresentamos a caracterização molecular da expressão distrofina 38 usando sequenciamento Sanger, RT-PCR e mancha ocidental no ensaio clínico.

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Medicina Edição 159 Medicina Distrofia muscular de Duchenne (DMD) Ensaio clínico Biópsia Muscular Primeiro em ensaio em humano Oligômeros morpholino (morfolinos ou PMOs) Distrófina Músculo Esquelético mRNA splicing RT-PCR inchaço ocidental NS-065/NCNP-01 exon 53
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