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Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In by 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
Published 2013Journal article -
2
Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides by 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
Published 2021Journal article -
3
GGPS1 mutations cause muscular dystrophy/hearing loss/ovarian insufficiency syndrome by 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
Published 2020Journal article