Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Titel:
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Auteur:
Kinali, Maria Arechavala-Gomeza, Virginia Feng, Lucy Cirak, Sebahattin Hunt, David Adkin, Carl Guglieri, Michela Ashton, Emma Abbs, Stephen Nihoyannopoulos, Petros Garralda, Maria Elena Rutherford, Mary Mcculley, Caroline Popplewell, Linda Graham, Ian R Dickson, George Wood, Matthew JA Wells, Dominic J Wilton, Steve D Kole, Ryszard Straub, Volker Bushby, Kate Sewry, Caroline Morgan, Jennifer E Muntoni, Francesco