Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-Deleted DMD Patient

• Takashi Saito,

  Affiliations Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan, Department of Pediatrics, School of Medicine, Tokyo Women's Medical University, Shinjuku, Tokyo, Japan

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• Akinori Nakamura,

  Affiliation Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan

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• Yoshitsugu Aoki,

  Affiliation Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan

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• Toshifumi Yokota,

  Affiliation Research Center for Genetic Medicine, Children's National Medical Center, Washington, District of Columbia, United States of America

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• Takashi Okada,

  Affiliation Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan

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• Makiko Osawa,

  Affiliation Department of Pediatrics, School of Medicine, Tokyo Women's Medical University, Shinjuku, Tokyo, Japan

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• Shin'ichi Takeda

  * E-mail: takeda@ncnp.go.jp

  Affiliation Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan

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