CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids
CRISPR-Cas9 校正 LCA5 中的无义突变可挽救人类视网膜类器官中 lebercilin 的表达和定位
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2023.05.012
Tess A V Afanasyeva, Dimitra Athanasiou, Pedro R L Perdigao, Kae R Whiting, Lonneke Duijkers, Galuh D N Astuti, Jean Bennett, Alejandro Garanto, Jacqueline van der Spuy, Ronald Roepman, Michael E Cheetham, Rob W J Collin