The human-specific RPGR isoform RPGRs14/15 and the clinically approved Rho/ROCK inhibitor Ripasudil represent therapeutic options to address RPGR-associated defects.
Muhammad Usman, Paul Atigbire, Dennis Kastrati, Julia Milena Brinkhoff, Charlotte Luise Kluth, Jannis Marticke, Christoph Jüschke, John Neidhardt
This study identifies the human-specific RPGR isoform RPGRs14/15 and the drug Ripasudil as potential therapies for RPGR-associated retinal degeneration by rescuing ciliary and actin defects in RPGR mutant cells.
bioRxiv · May 9, 2025 · PreprintCell Biology