Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In por Popplewell, L, Koo, T, Leclerc, X, Duclert, A, Mamchaoui, K, Gouble, A, Mouly, V, Voit, T, Pâques, F, Cédrone, F, Isman, O, Yáñez-Muñoz, R, Dickson, G

Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides por Tone, Y, Mamchaoui, K, Tsoumpra, MK, Hashimoto, Y, Terada, R, Maruyama, R, Gait, MJ, Arzumanov, AA, McClorey, G, Imamura, M, Takeda, S, Yokota, T, Wood, MJA, Mouly, V, Aoki, Y

GGPS1 mutations cause muscular dystrophy/hearing loss/ovarian insufficiency syndrome por Foley, AR, Zou, Y, Dunford, JE, Rooney, J, Chandra, G, Xiong, H, Straub, V, Voit, T, Romero, N, Donkervoort, S, Hu, Y, Markello, T, Horn, A, Qebibo, L, Dastgir, J, Meilleur, KG, Finkel, RS, Fan, Y, Mamchaoui, K, Duguez, S, Nelson, I, Laporte, J, Santi, M, Malfatti, E, Maisonobe, T, Touraine, P, Hirano, M, Hughes, I, Bushby, K, Oppermann, U, Böhm, J, Jaiswal, JK, Stojkovic, T, Bönnemann, CG