Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoids
利用腺嘌呤碱基编辑器校正患者iPSC和视网膜类器官中与遗传性视网膜营养不良相关的致病变异
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102777
Leung, Amy; Perdigão, Pedro R L; Sacristan-Reviriego, Almudena; Sladen, Paul E; Aguzzi, Erika A; Rezek, Farah O; Ziaka, Kalliopi; Guarascio, Rosellina; Hau, Kwan-Leong; Cheetham, Michael E; van der Spuy, Jacqueline
