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 journalArticlepeer-review

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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

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