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Dose finding study in the DMDmdx rat model to determine the efficacious dose of a rAAV9 vector encoding a human mini-dystrophin after IV administration

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Le Guiner, Caroline | Mclntyre, M | Larcher, Thibaut | Adjali, Oumeya | Lafoux, Aude | Toumaniantz, Gilles | Wood, L | Xiao, X | Moullier, Philippe | Samulski, R

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Dose finding study in the DMDmdx rat model to determine the efficacious dose of a rAAV9 vector encoding a human mini-dystrophin after IV administration. 22nd International Annual Congress of the World-Muscle-Society (WMS)

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