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  1. 1
    Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells

    Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells by Rachele Rossi, Silvia Torelli, Marc Moore, Pierpaolo Ala, Jennifer Morgan, Jyoti Malhotra, Francesco Muntoni

    Published 2024-11-01
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  2. 2
    Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next‐generation human artificial chromosomes for Duchenne muscular dystrophy

    Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next‐generation human artificial chromosomes for Duchenne muscular dystrophy by Sara Benedetti, Narumi Uno, Hidetoshi Hoshiya, Martina Ragazzi, Giulia Ferrari, Yasuhiro Kazuki, Louise Anne Moyle, Rossana Tonlorenzi, Angelo Lombardo, Soraya Chaouch, Vincent Mouly, Marc Moore, Linda Popplewell, Kanako Kazuki, Motonobu Katoh, Luigi Naldini, George Dickson, Graziella Messina, Mitsuo Oshimura, Giulio Cossu, Francesco Saverio Tedesco

    Published 2017-12-01
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