Therapeutic approaches to muscular dystrophy von Goyenvalle, Aurélie, Seto, Jane T, Davies, Kay E, Chamberlain, Jeffrey

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Live‐imaging of revertant and therapeutically restored dystrophin in the DmdEGFP‐mdx mouse model for Duchenne muscular dystrophy von Petkova, M. V., Stantzou, A., Morin, A., Petrova, O., Morales‐Gonzalez, S., Seifert, F., Bellec‐Dyevre, J., Manoliu, T., Goyenvalle, A., Garcia, L., Richard, I., Laplace‐Builhé, C., Schuelke, M., Amthor, H.

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Live-imaging of revertant and therapeutically restored dystrophin in the Dmd EGFP-mdx mouse model for Duchenne muscular dystrophy von Petkova, M V, Stantzou, A, Morin, A, Petrova, O, Morales-Gonzalez, S, Seifert, F, Bellec-Dyevre, J, Manoliu, T, Goyenvalle, A, Garcia, L, Richard, I, Laplace-Builhé, C, Schuelke, M, Amthor, H

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O09 Tricyclo-DNA for the treatment of neuromuscular diseases von Goyenvalle, A, Robin, V, Garcia, L, Dugovic, B, Leumann, C.J

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Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers von Goyenvalle, Aurélie, Griffith, Graziella, Babbs, Arran, Andaloussi, Samir El, Ezzat, Kariem, Avril, Aurélie, Dugovic, Branislav, Chaussenot, Rémi, Ferry, Arnaud, Voit, Thomas, Amthor, Helge, Bühr, Claudia, Schürch, Stefan, Wood, Matthew J A, Davies, Kay E, Vaillend, Cyrille, Leumann, Christian, Garcia, Luis

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T.O.3 Tricyclo-DNA: A promising chemistry for the synthesis of antisense molecules for splice-switching approaches in DMD von Goyenvalle, A, Babbs, A, Avril, A, Griffiths, G, Dugovic, B, Davies, K.E, Leumann, C, Garcia, L

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P.20.8 AAV genome loss from dystrophic mouse muscles during AAV-U7snRNA-mediated exon skipping therapy von Hir, M. Le, Goyenvalle, A, Peccate, C, Précigout, G, Davies, K.E, Voit, T, Garcia, L, Lorain, S

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P3.02 AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy von Goyenvalle, A, Babbs, A, Garcia, L, Davies, K.E

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Rescue of Dystrophic Muscle through U7 snRNA-Mediated Exon Skipping von Goyenvalle, Aurélie, Vulin, Adeline, Fougerousse, Françoise, Leturcq, France, Kaplan, Jean-Claude, Garcia, Luis, Danos, Olivier

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P06 AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy von Goyenvalle, A, Babbs, A, Wright, J, Garcia, L, Davies, K.E

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249th ENMC International Workshop: The role of brain dystrophin in muscular dystrophy: Implications for clinical care and translational research, Hoofddorp, The Netherlands, Novemb... von Hendriksen, Jos G.M., Thangarajh, Mathula, Kan, Hermien E., Muntoni, Francesco, Aoki, Dr. Y., Collin, Dr P., Colvin, Dr M., Doorenweerd, Dr N., Ferlini, Prof A., Goyenvalle, Dr A., Hendriksen, Dr J.J., Hoskin, Dr J., Kan, Dr H.E., Lamy, Mr F., Maresh, Dr K., Muntoni, Prof F., Niks, Dr E.H., Schara, Prof U., Skuse, Prof D., Straub, Prof V., Takeda, Prof S., Thangarajh, Dr M., Truba, Dr N., Tyagarajan, Prof S., Vaillend, Dr C., van Putten, Dr M., Vissing, Prof J., Vroom, E.

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Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies von BARTOLI, M, POUPIOT, J, GOYENVALLE, A, PEREZ, N, GARCIA, L, DANOS, O, RICHARD, I

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306P Evaluating genetic therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics von Mao, Q., Ahmadi, A., van Doorne, L., de Vries, S., Heezen, L., Vacca, O., Aartsma-Rus, A., van Putten, M., Goyenvalle, A., Mahfouz, A., Spitali, P.

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Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides von Ezzat, Kariem, Aoki, Yoshitsugu, Koo, Taeyoung, McClorey, Graham, Benner, Leif, Coenen-Stass, Anna, O’Donovan, Liz, Lehto, Taavi, Garcia-Guerra, Antonio, Nordin, Joel, Saleh, Amer F, Behlke, Mark, Morris, John, Goyenvalle, Aurelie, Dugovic, Branislav, Leumann, Christian, Gordon, Siamon, Gait, Michael J, El−Andaloussi, Samir, Wood, Matthew JA

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P26 Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping von Goyenvalle, A, Babbs, A, Powell, D, Kole, R, Fletcher, S, Wilton, S.D, Davies, K.E

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300P Brain involvement in Duchenne muscular dystrophy (BIND) von Tetorou, K., Aghaeipour, A., Gileadi, T., Ma, S., Quinteiro, P. Perdomo, Saoudi, A., Ceschi, L., Zarrouki, F., Mitsogiannis, M., Fergus, C., Kelly, V., Zhang, L., Spitali, P., Aoki, Y., Morgan, J., Montanaro, F., Vaillend, C., Goyenvalle, A., Muntoni, F.

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P.301 - The relationship between dystrophin restoration through tricyclo DNA-AON treatment to myosin fast fibre phenotype and other members of the DAG complex von Omairi, S., Goyenvalle, A., Amthor, H., Garcia, L., Patel, K.

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The relationship between dystrophin restoration through tricyclo DNA-AON treatment to myosin fast fibre phenotype and other members of the DAG complex von Omairi, S., Goyenvalle, A., Amthor, H., Garcia, L., Patel, K.

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DMD – ANIMAL MODELS von Morin, A., Petrova, O., Petkova, M., Tensorer, T., Manoliu, T., Richard, I., Garcia, L., Schuelke, M., Laplace-Builhé, C., Goyenvalle, A., Stantzou, A., Amthor, H.

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DMD – ANIMAL MODELS: EP.77 Subcellular organization of skeletal muscle dystrophin is restored by tricyclo-DNA mediated exon skipping in the DmdEGFP-mdx mouse model von Morin, A., Petrova, O., Petkova, M., Tensorer, T., Manoliu, T., Richard, I., Garcia, L., Schuelke, M., Laplace-Builhé, C., Goyenvalle, A., Stantzou, A., Amthor, H.

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