Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

Por Lu-Nguyen, Ngoc B, Jarmin, Susan A, Saleh, Amer F, Popplewell, Linda, Gait, Michael J, Dickson, George
Publicado no Mol Ther (2015)

PABPN1 gene therapy for oculopharyngeal muscular dystrophy

Por Malerba, A., Klein, P., Bachtarzi, H., Jarmin, S. A., Cordova, G., Ferry, A., Strings, V., Espinoza, M. Polay, Mamchaoui, K., Blumen, S. C., St Guily, J. Lacau, Mouly, V., Graham, M., Butler-Browne, G., Suhy, D. A., Trollet, C., Dickson, G.
Publicado no Nat Commun (2017)