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    Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA

    Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA by Paolo Peruzzo, Natascha Bergamin, Martina Bon, Sara Cappelli, Alessandra Longo, Elisa Goina, Cristiana Stuani, Emanuele Buratti, Andrea Dardis

    Published 2025-02-01
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