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EXON SKIPPING AND DYSTROPHIN RESTORATION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS AFTER SYSTEMIC PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT
Presentation / Conference Contribution
Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Garralda, M. E., Wells, D. J., Dickson, G., Wood, M. J. A., Wilton, S. D., Straub, V., Shrewsbury, S. B., Sewry, C., Morgan, J. E., Bushby, K., & Muntoni, F. EXON SKIPPING AND DYSTROPHIN RESTORATION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS AFTER SYSTEMIC PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-esclation study
Journal Article
Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Abbs, S., Garralda, M. E., Wells, D., Dickson, G., Wood, M. J. A., Wilton, S. D., Straub, V., Shrewsbury, S., Sewry, C., Morgan, J. E., 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-esclation study. https://doi.org/10.1016/S0140-6736%2811%2960756-3

Molecular Mechanism of the E99K Mutation in Cardiac Actin (ACTC Gene) That Causes Apical Hypertrophy in Man and Mouse
Journal Article
Song, W., Dyer, E., Stuckey, D. J., Copeland, O., Leung, M. C., Bayliss, C., Messer, A., Wilkinson, R., Tremoleda, J. L., Schneider, M. D., Harding, S. E., Redwood, C. S., Clarke, K., Nowak, K., Monserrat, L., Wells, D. J., & Marston, S. B. (in press). Molecular Mechanism of the E99K Mutation in Cardiac Actin (ACTC Gene) That Causes Apical Hypertrophy in Man and Mouse. Journal of Biological Chemistry, 286(31), 27582–27593. https://doi.org/10.1074/jbc.M111.252320