Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice
Forand A., Muchir A., Mougenot N., Sevoz-Couche C., Peccate C., Lemaitre M., Izabelle C., Wood M., Lorain S., Piétri-Rouxel F.
© 2020 The Authors The AAV-based gene therapies used in DMD have the major limitation of maintaining the therapeutic effect over time. Forand et al. demonstrated an undeniable advantage of the PPMO and AAV combination on the striated muscles, notably on heart and diaphragm structure and function with an unrivalled extent of survival.