Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knock-down and replacement of mutant expanded PABPN1. / Malerba, Alberto; Klein, Pierre; Lu-Nguyen, Ngoc; Cappellari, Ornella; Strings-Ufombah, Vanessa; Harbaran, Sonal; Roelvink, Peter; Suhy, David; Trollet, Capucine; Dickson, George.

In: Human Molecular Genetics, 01.10.2019, p. 1-24.

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Original languageEnglish
Article numberddz167
Pages (from-to)1-24
Number of pages24
JournalHuman Molecular Genetics
Early online date11 Jul 2019
DOIs
Publication statusPublished - 1 Oct 2019
This open access research output is licenced under a Creative Commons Attribution-NonCommercial-NoDerivs 3.0 Unported License.

ID: 34308878