1. Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1. (2nd July 2019) Authors: Malerba, Alberto; Klein, Pierre; Lu-Nguyen, Ngoc; Cappellari, Ornella; Strings-Ufombah, Vanessa; Harbaran, Sonal; Roelvink, Peter; Suhy, David; Trollet, Capucine; Dickson, George Journal: Human molecular genetics Issue: Volume 28:Number 19(2019) Page Start: 3301 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗