Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knock-down and replacement of mutant expanded PABPN1

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.

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