Gene Supplementation of MYO7A or activation of Myo7b for treatment of Usher syndrome 1B
David Manuel Mittas, Dina Yehia Otify, Zoran Gavrilov, Thomas Heigl, Jan Suchomski, Paulina Deltuvaite, Klara Sonnie Hinrichsmeyer, Olivier Mercey, Franz Kynast, Jan Motlík, Zdeňka Ellederová, Taras Ardan, Andreas Klingl, Jennifer Grünert, Verena Mehlfeld, , Elvir Bećirović
This study evaluates gene supplementation of MYO7A via dual AAVs and CRISPR/Cas activation of Myo7b in animal models and human retinal organoids to treat Usher syndrome 1B.
bioRxiv · Jul 3, 2026 · PreprintMolecular Biology