Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice
肽偶联磷酰二胺吗啉寡聚体(PPMO)与AAV-U7联合治疗可挽救小鼠的严重DMD表型
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2020.03.011
Forand, Anne; Muchir, Antoine; Mougenot, Nathalie; Sevoz-Couche, Caroline; Peccate, Cécile; Lemaitre, Mégane; Izabelle, Charlotte; Wood, Matthew; Lorain, Stéphanie; Piétri-Rouxel, France