Targeting lysosomal damage is a new therapeutic perspective for Duchenne Muscular Dystrophy
Abbass Jaber, Laura Palmieri, Rania Bakour, Elise Lachiver, Ai Vu Hong, Nathalie Bourg, Jérôme Poupiot, Sonia Albini, Daniel Stockholm, Laetitia van Wittenberghe, Adeline Miranda, Nathalie Danièle, Inès Barthélémy, Stéphane Blot, Mai Thao Bui, , David Israeli
This study identifies lysosomal damage as a novel mechanism in Duchenne Muscular Dystrophy and shows that combining trehalose with gene therapy improves outcomes.
bioRxiv · Feb 11, 2025 · PreprintPhysiology