Exon skipping and dystrophin restoration in Duchenne muscular dystrophy patients after systemic phosphorodiamidate morpholino oligomer treatment

Sebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, Lucy Feng, Silvia Torelli, Karen Anthony, Maria Elena Garralda, Dominic J. Wells, George Dickson, Matthew Ja Wood, Stephen D. Wilton, Volker Straub, Stephen B. Shrewsbury, Caroline Sewry, Jennifer Morgan, Kate Bushby, Francesco Muntoni

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Medicine & Life Sciences