Clinically translatable mitochondrial gene therapy in muscle using tandem mtZFN architecture.
利用串联 mtZFN 结构在肌肉中进行临床转化线粒体基因治疗
期刊:EMBO Molecular Medicine
影响因子:8.3
doi:10.1038/s44321-025-00231-5
Nash Pavel A, Turner Keira M, Powell Christopher A, Van Haute Lindsey, Silva-Pinheiro Pedro, Bubeck Felix, Wiedtke Ellen, Marques Eloïse, Ryan Dylan G, Grimm Dirk, Gammage Payam A, Minczuk Michal
