Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy
Shouta Miyatake, Yoshitaka Mizobe, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Shin’ichi Takeda, Yoshitsugu Aoki
Abstract
Shouta Miyatake, Yoshitaka Mizobe, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Shin’ichi Takeda, Yoshitsugu Aoki
Abstract
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Key concepts: Exon skipping, Morpholino, Duchenne muscular dystrophy, Dystrophin, Exon, mdx mouse, RNA splicing, Muscular dystrophy