Expanding the options for therapeutic exon skipping as a future treatment for USH2A-associated disease by 3D structural modeling of newly formed hybrid domains
Lucija Malinar, Sanne Broekman, Daniel T. Rademaker, Anh Q Le, Theo A. Peters, Erik de Vrieze, Peter A. C. 't Hoen, Erwin van Wijk, Hanka Venselaar
This study uses AlphaFold2 to predict viable hybrid domains from exon skipping combinations in USH2A, expanding therapeutic options for Usher syndrome.
bioRxiv · Apr 28, 2026 · PreprintBioinformatics