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

Maria Kinali, Virginia Arechavala-Gomeza, Lucy Feng, Sebahattin Cirak, David Hunt, Carl Adkin, Michela Guglieri, Emma Ashton, Stephen Abbs, Petros Nihoyannopoulos, Maria Elena Garralda, Mary Rutherford, Caroline McCulley, Linda Popplewell, Ian R Graham, George Dickson, Matthew J A Wood, Dominic J Wells, Steve D Wilton, Ryszard KoleVolker Straub, Kate Bushby, Caroline Sewry, Jennifer E Morgan, Francesco Muntoni

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Medicine and Dentistry

Neuroscience

Biochemistry, Genetics and Molecular Biology