RNA approaches for treatment of SMA in human motor neurons

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Jon Moulton
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RNA approaches for treatment of SMA in human motor neurons

Nizzardo M, Simone C, Dametti S, Salani S, Ulzi G, Pagliarani S, Rizzo F, Frattini E, Pagani F, Bresolin N, Comi G, Corti S. Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches. Sci Rep. 2015 Jun 30;5:11746. doi: 10.1038/srep11746.