Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping. by Goyenvalle, A, Babbs, A, Powell, D, Kole, R, Fletcher, S, Wilton, S, Davies, K

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping by Goyenvalle, A, Babbs, A, Powell, D, Kole, R, Fletcher, S, Wilton, S, Davies, K

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-... by Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K, Abbs, S, Garralda, M, Bourke, J, Wells, D, Dickson, G, Wood, M, Wilton, S, Straub, V, Kole, R, Shrewsbury, S, Sewry, C, Morgan, J, Bushby, K, Muntoni, F

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-... by Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K, Abbs, S, Garralda, M, Bourke, J, Wells, D, Dickson, G, Wood, M, Wilton, S, Straub, V, Kole, R, Shrewsbury, S, Sewry, C, Morgan, J, Bushby, K, Muntoni, F

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-conce... by Kinali, M, Arechavala-Gomeza, V, Feng, L, Cirak, S, Hunt, D, Adkin, C, Guglieri, M, Ashton, E, Abbs, S, Nihoyannopoulos, P, Garralda, M, Rutherford, M, McCulley, C, Popplewell, L, Graham, I, Dickson, G, Wood, M, Wells, D, Wilton, S, Kole, R, Straub, V, Bushby, K, Sewry, C, Morgan, J, Muntoni, F