Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.

"Declarations of interests J.O., H.N., and V.F. are employees of and hold stock in Pfizer. C.L.G. and P.M. are co-authors of a patent for systemic treatment of dystrophic pathologies (EP3044319A1, dated June 27, 2014)."

"This study was sponsored by 10.13039/100004319Pfizer. We thank all personnel of the Boisbonne Center for Gene Therapy (ONIRIS, INSERM, Nantes, France) for handling and care of the rats included in this study. We also thank the staff of the Preclinical Analytics Core (TaRGeT lab, Nantes Université, CHU Nantes, INSERM, Nantes, France), the Gene Therapy Immunology Core (TaRGeT lab, Nantes Université, CHU Nantes, INSERM, Nantes, France), the Therassay Core (Capacités, Nantes Université, France), and the APEX Core (INRAE, ONIRIS, Nantes, France) for technical assistance. We thank the MDA Monoclonal Antibody Resource for providing the MANEX 1011C antibody. Medical writing support was provided by Vardit Dror, PhD; Charles S. Cheng; and David Cope, PhD, of Engage Scientific Solutions and was funded by 10.13039/100004319Pfizer."