Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy
优化碱基编辑器,用于功能性校正 SMN2 以治疗脊髓性肌萎缩症
期刊:Nature Biomedical Engineering
影响因子:26.8
doi:10.1038/s41551-023-01132-z
Christiano R R Alves, Leillani L Ha, Rebecca Yaworski, Emma R Sutton, Cicera R Lazzarotto, Kathleen A Christie, Aoife Reilly, Ariane Beauvais, Roman M Doll, Demitri de la Cruz, Casey A Maguire, Kathryn J Swoboda, Shengdar Q Tsai, Rashmi Kothary, Benjamin P Kleinstiver