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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides.
Nordin, Joel Z; Mizobe, Yoshitaka; Nakamura, Harumasa; Komaki, Hirofumi; Takeda, Shin'ichi; Aoki, Yoshitsugu.
Afiliación
  • Nordin JZ; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry.
  • Mizobe Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry.
  • Nakamura H; Clinical Research Support Office, Translational Medical Center, National Center of Neurology and Psychiatry.
  • Komaki H; Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry.
  • Takeda S; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry.
  • Aoki Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry; tsugu56@ncnp.go.jp.
J Vis Exp ; (159)2020 05 07.
Article en En | MEDLINE | ID: mdl-32955503
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Exones / Ensayos Clínicos como Asunto / Oligonucleótidos Antisentido / Distrofia Muscular de Duchenne Límite: Humans Idioma: En Revista: J Vis Exp Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Exones / Ensayos Clínicos como Asunto / Oligonucleótidos Antisentido / Distrofia Muscular de Duchenne Límite: Humans Idioma: En Revista: J Vis Exp Año: 2020 Tipo del documento: Article Pais de publicación: Estados Unidos