Splicing modulation therapy in the treatment of genetic diseases by Arechavala-Gomeza V, Khoo B, Aartsma-Rus A

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The Pathogenesis and Therapy of Muscular Dystrophies by Guiraud, S, Aartsma-Rus, A, Vieira, N, Davies, K, van Ommen, G, Kunkel, L

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD by Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J

Deacetylation inhibition reverses PABPN1-dependent muscle wasting by Olie, C, Riaz, M, Konietzny, R, Charles, P, Pinto-Fernandez, A, Kielbasa, S, Aartsma-Rus, A, Goeman, J, Kessler, B, Raz, V

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. by Pichavant, C, Aartsma-Rus, A, Clemens, P, Davies, K, Dickson, G, Takeda, S, Wilton, S, Wolff, J, Wooddell, C, Xiao, X, Tremblay, J

Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology by Van Westering, T, Lomonosova, Y, Coenen‐Stass, A, Betts, C, Bhomra, A, Hulsker, M, Clark, L, McClorey, G, Aartsma‐Rus, A, Putten, M, Wood, M, Roberts, T

Delivery is key: lessons learnt from developing splice-switching antisense therapies by Godfrey, C, Desviat, L, Smedsrød, B, Piétri-Rouxel, F, Denti, M, Disterer, P, Lorain, S, Nogales-Gadea, G, Sardone, V, Anwar, R, El Andaloussi, S, Lehto, T, Khoo, B, Brolin, C, , v, Goyenvalle, A, Aartsma-Rus, A, Arechavala-Gomeza, V

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities by Hammond, SM, Aartsma‐Rus, A, Alves, S, Borgos, SE, Buijsen, RAM, Collin, RWJ, Covello, G, Denti, MA, Desviat, LR, Echevarría, L, Foged, C, Gaina, G, Garanto, A, Goyenvalle, AT, Guzowska, M, Holodnuka, I, Jones, DR, Krause, S, Lehto, T, Montolio, M, Van Roon‐Mom, W, Arechavala‐Gomeza, V

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy by Straub, V, Balabanov, P, Bushby, K, Ensini, M, Goemans, N, De Luca, A, Pereda, A, Hemmings, R, Campion, G, Kaye, E, Arechavala-Gomeza, V, Goyenvalle, A, Niks, E, Veldhuizen, O, Furlong, P, Stoyanova-Beninska, V, Wood, M, Johnson, A, Mercuri, E, Muntoni, F, Sepodes, B, Haas, M, Vroom, E, Aartsma-Rus, A