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

Alberto Malerba, Pierre Klein, Ngoc Lu-Nguyen, Ornella Cappellari, Vanessa Strings-Ufombah, Sonal Harbaran, Peter Roelvink, David Suhy, Capucine Trollet, George Dickson

Research output: Contribution to journal › Article › peer-review

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