Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy. by Goyenvalle, A, Davies, K

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy. by Goyenvalle, A, Davies, K

Therapeutic approaches to muscular dystrophy. by Goyenvalle, A, Seto, J, Davies, K, Chamberlain, J

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy by Goyenvalle, A, Babbs, A, Garcia, L, Davies, K

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients by Goyenvalle, A, Babbs, A, Garcia, L, Davies, K

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

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy. by Goyenvalle, A, Babbs, A, van Ommen, G, Garcia, L, Davies, K

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery. by Eckenfelder, A, Tordo, J, Babbs, A, Davies, K, Goyenvalle, A, Danos, O

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy by Goyenvalle, A, Wright, J, Babbs, A, Wilkins, V, Garcia, L, Davies, K

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping. by Goyenvalle, A, Babbs, A, Powell, D, Kole, R, Fletcher, S, Wilton, S, Davies, K

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy. by Goyenvalle, A, Wright, J, Babbs, A, Wilkins, V, Garcia, L, Davies, K

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

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping by Goyenvalle, A, Babbs, A, Wright, J, Wilkins, V, Powell, D, Garcia, L, Davies, K

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping. by Goyenvalle, A, Babbs, A, Wright, J, Wilkins, V, Powell, D, Garcia, L, Davies, K

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy by Le Hir, M, Goyenvalle, A, Peccate, C, Précigout, G, Davies, K, Voit, T, Garcia, L, Lorain, S

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

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice. by Crisp, A, Yin, H, Goyenvalle, A, Betts, C, Moulton, H, Seow, Y, Babbs, A, Merritt, T, Saleh, A, Gait, M, Stuckey, D, Clarke, K, Davies, K, Wood, M

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

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