EXON SKIPPING AND DYSTROPHIN RESTORATION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS AFTER SYSTEMIC PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT
Journal Article
Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., …Muntoni, F. EXON SKIPPING AND DYSTROPHIN RESTORATION IN DUCHENNE MUSCULAR DYSTROPHY PATIENTS AFTER SYSTEMIC PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER TREATMENT. Neuromuscular Disorders, 21(1), S7-S8
Outputs (83)
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., …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
Influence of needle gauge on in vivo ultrasound and microbubble-mediated gene transfection
Journal Article
Browning, R. J., Mulvana, H., Tang, M. X., Hajnal, J. V., Wells, D. J., & Eckersley, R. J. Influence of needle gauge on in vivo ultrasound and microbubble-mediated gene transfection. Ultrasound in Medicine and Biology, 37(9), 1531-1537. https://doi.org/10.1016/j.ultrasmedbio.2011.05.019
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., …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
ASSESSING THE LONG TERM EXPRESSION OF LARGE AS A POTENTIAL THERAPY IN A MOUSE MODEL OF LGMD2I
Journal Article
Whitmore, C., Booler, H., Parr, C., Ackroyd, M. R., Fernandez-Fuente, M., Muntoni, F., …Brown, S. C. ASSESSING THE LONG TERM EXPRESSION OF LARGE AS A POTENTIAL THERAPY IN A MOUSE MODEL OF LGMD2I. Neuromuscular Disorders, 22(9-10), 813-814. https://doi.org/10.1016/j.nmd.2012.06.040
OVER EXPRESSION OF THE LARGE TRANSGENE EXACERBATES MUSCLE PATHOLOGY IN THE MDX MOUSE
Journal Article
Wells, K. E., Muses, S., Ruberg, A., Terry, R., Shihab, N. K., & Wells, D. J. OVER EXPRESSION OF THE LARGE TRANSGENE EXACERBATES MUSCLE PATHOLOGY IN THE MDX MOUSE. Neuromuscular Disorders, 22(9-10), 813-813. https://doi.org/10.1016/j.nmd.2012.06.039
TREATMENT WITH ORAL METFORMIN IMPROVES MUSCLE FUNCTION IN MDX MICE
Journal Article
Terry, R. L., & Wells, D. J. TREATMENT WITH ORAL METFORMIN IMPROVES MUSCLE FUNCTION IN MDX MICE. Neuromuscular Disorders, 22(9-10), 847-847. https://doi.org/10.1016/j.nmd.2012.06.150
POLOXOMER 188 HAS A DELETERIOUS EFFECT ON SKELETAL MUSCLE FUNCTION IN THE MDX MOUSE
Journal Article
Terry, R. L., Kaneb, H. M., & Wells, D. J. POLOXOMER 188 HAS A DELETERIOUS EFFECT ON SKELETAL MUSCLE FUNCTION IN THE MDX MOUSE. Neuromuscular Disorders, 22, S14-S14
Heat shock protein 27 protects against atherogenesis via an Estrogen-Dependent Mechanism: Role of Selective Estrogen Receptor Beta Modulation
Journal Article
Rayner, K., Sun, J., Chen, Y. X., McNulty, M., Simard, T., Zhao, X., …O'Brien, E. R. Heat shock protein 27 protects against atherogenesis via an Estrogen-Dependent Mechanism: Role of Selective Estrogen Receptor Beta Modulation. Arteriosclerosis, Thrombosis, and Vascular Biology, 29(11), 1751-6. https://doi.org/10.1161/ATVBAHA.109.193656
INVESTIGATING THE EFFECTS OF DAO TRANSGENES ON THE SOD1(G93A) MOUSE MODEL OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
Journal Article
Rahmani-Kondori, N., de Belleroche, J., & Wells, D. J. INVESTIGATING THE EFFECTS OF DAO TRANSGENES ON THE SOD1(G93A) MOUSE MODEL OF AMYOTROPHIC LATERAL SCLEROSIS (ALS). Neuromuscular Disorders, 22, S13-S13