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Exon skipping and dystrophin restoration in patients with duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

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Lentivirus of the Brunello CRISPR library was produced in HEK293T cells by co-transfection of the sgRNA pooled library in LentiCRISPRv2 plasmid together with pMD2.G and psPAX lentiviral helper plasmids using Xfect as the transfection reagent in T225 flasks 17

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