Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice
对患者造血干细胞进行体外主要编辑可在小鼠植入后挽救镰状细胞病表型
期刊:Nature Biomedical Engineering
影响因子:26.8
doi:10.1038/s41551-023-01026-0
Kelcee A Everette, Gregory A Newby, Rachel M Levine, Kalin Mayberry, Yoonjeong Jang, Thiyagaraj Mayuranathan, Nikitha Nimmagadda, Erin Dempsey, Yichao Li, Senthil Velan Bhoopalan, Xiong Liu, Jessie R Davis, Andrew T Nelson, Peter J Chen, Alexander A Sousa, Yong Cheng, John F Tisdale, Mitchell J Weis