Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice

Anne Forand(Inserm), France Piétri‐Rouxel(Inserm), Charlotte Izabelle(Centre National de la Recherche Scientifique), Caroline Sévoz‐Couche(Inserm), Nathalie Mougenot(Inserm), Cécile Peccate(Inserm), Matthew J. A. Wood(University of Oxford), Antoine Muchir(Inserm), Mégane Lemaître(Inserm), Stéphanie Lorain(Inserm)
Molecular Therapy — Methods & Clinical Development
March 17, 2020
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