A Malerba
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
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 |