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

Malerba, A; Klein, P; Lu-Nguyen, N; Cappellari, O; Strings-Ufombah, V; Harbaran, S; Roelvink, P; Suhy, D; Trollet, C; Dickson, G

Authors

A Malerba

P Klein

N Lu-Nguyen

O Cappellari

V Strings-Ufombah

S Harbaran

P Roelvink

D Suhy

C Trollet

G Dickson



Citation

Malerba, A., Klein, P., Lu-Nguyen, N., Cappellari, O., Strings-Ufombah, V., Harbaran, S., …Dickson, G. (in press). 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, 28(19), 3301-3308. https://doi.org/10.1093/hmg/ddz167

Journal Article Type Article
Acceptance Date Jul 8, 2019
Deposit Date Aug 1, 2019
Journal HUMAN MOLECULAR GENETICS
Print ISSN 0964-6906
Publisher Oxford University Press
Volume 28
Issue 19
Pages 3301-3308
DOI https://doi.org/10.1093/hmg/ddz167
Public URL https://rvc-repository.worktribe.com/output/1381334

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