A human-specific RPGR isoform and a clinically approved Rho/ROCK inhibitor ameliorate defects associated with RPGR dysfunction
人源特异性RPGR亚型和临床批准的Rho/ROCK抑制剂可改善与RPGR功能障碍相关的缺陷。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102758
Usman, Muhammad; Atigbire, Paul; Kastrati, Dennis; Brinkhoff, Julia Milena; Kluth, Charlotte Luise; Marticke, Jannis; Jüschke, Christoph; Neidhardt, John
