CRISPR/Cas13-mediated Dynamin 2 reduction therapy in a canine model of DNM2-related centronuclear myopathy
Andrea Carlier, Inès Barthélémy, Nicolas Blanchard‐Gutton, Sophie Château‐Joubert, Frédéric Aurade, Teoman Ozturk, Nathalie Didier, Frédéric Relaix, Laurent Tiret, Stéphane Blot, Isabel Punzón
This study demonstrates that CRISPR/Cas13-mediated reduction of DNM2 expression can alleviate CNM symptoms in a canine model.
bioRxiv · Sep 5, 2025 · PreprintMolecular Biology