AAV-mediated knock-down and replacement of mutant expanded PABPN 1

1 Centres of Gene and Cell Therapy and Biomedical Sciences, School of Biological Sciences, Royal Holloway University of London, Egham, Surrey, UK 2 Sorbonne Université, INSERM, Association Institut de Myologie, Centre de Recherche en Myologie, UMRS974, 47 bd de l’Hôpital, 75013 Paris, France 3 Comparative Biomedical Sciences, Royal Veterinary College, London, UK 4 Benitec Biopharma, Hayward, California, USA