Guanabenz treatment improves Oculopharyngeal muscular dystrophy phenotype
Alberto Malerba, Fanny Roth, Pradeep Harish, Jamila Dhiab, Ngoc Lu-Nguyen, Ornella Cappellari, Susan Jarmin, Alexandrine Mahoudeau, Victor Ythier, Jeanne Lainé, Elisa Negroni, Emmanuelle Abgueguen, Martine Simonelig, Philippe Guedat, Vincent Mouly, , Capucine Trollet
This study shows that guanabenz acetate reduces PABPN1 aggregates and improves muscle function in an OPMD mouse model by modulating the UPR.
bioRxiv · Jul 24, 2018 · PreprintPathology