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Malerba, A and Klein, P and Lu-Nguyen, N and Cappellari, O and Strings-Ufombah, V and Harbaran, S and Roelvink, P and Suhy, D and Trollet, C and Dickson, G (2019) Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knock-down and replacement of mutant expanded PABPN1. HUMAN MOLECULAR GENETICS.
Harish, P and Malerba, A and Lu-Nguyen, N and Forrest, L and Cappellari, O and Roth, F and Trollet, C and Popplewell, L and Dickson, G (2019) Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD). Journal of Cachexia, Sarcopenia and Muscle.
Malerba, A and Roth, F and Harish, P and Dhiab, J and Lu-Nguyen, N and Cappellari, O and Jarmin, S and Mahoudeau, A and Ythier, V and Lainé, J and Negroni, E and Abgueguen, E and Simonelig, M and Guedat, P and Mouly, V and Butler-Browne, G and Voisset, C and Dickson, G and Trollet, C (2019) Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy. HUMAN MOLECULAR GENETICS.