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) ↗
2. Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice. (10th June 2019) Authors: Lu-Nguyen, Ngoc; Ferry, Arnaud; Schnell, Frederick J; Hanson, Gunnar J; Popplewell, Linda; Dickson, George; Malerba, Alberto Journal: Human molecular genetics Issue: Volume 28:Number 18(2019) Page Start: 3091 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. G-quadruplex ligands mediate downregulation of DUX4 expression. Issue 8 (17th March 2020) Authors: Ciszewski, Lukasz; Lu-Nguyen, Ngoc; Slater, Alex; Brennan, Andrew; Williams, Huw E L; Dickson, George; Searle, Mark S; Popplewell, Linda Journal: Nucleic acids research Issue: Volume 48:Issue 8(2020) Page Start: 4179 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy. (14th January 2019) Authors: Malerba, Alberto; Roth, Fanny; Harish, Pradeep; Dhiab, Jamila; Lu-Nguyen, Ngoc; Cappellari, Ornella; Jarmin, Susan; Mahoudeau, Alexandrine; Ythier, Victor; Lainé, Jeanne; Negroni, Elisa; Abgueguen, Emmanuelle; Simonelig, Martine; Guedat, Philippe; Mouly, Vincent; Butler-Browne, Gillian; Voisset, ... Journal: Human molecular genetics Issue: Volume 28:Number 10(2019) Page Start: 1694 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model. Issue 15 (13th May 2021) Authors: Lu-Nguyen, Ngoc; Malerba, Alberto; Herath, Shan; Dickson, George; Popplewell, Linda Journal: Human molecular genetics Issue: Volume 30:Issue 15(2021) Page Start: 1398 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗