Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations

por Woodard, Kaitly J., Doerfler, Phillip A., Mayberry, Kalin D., Sharma, Akshay, Levine, Rachel, Yen, Jonathan, Valentine, Virginia, Palmer, Lance E., Valentine, Marc, Weiss, Mitchell J.
Publicado 2022

An RPS19-edited model for Diamond-Blackfan anemia reveals TP53-dependent impairment of hematopoietic stem cell activity

por Bhoopalan, Senthil Velan, Yen, Jonathan S., Mayuranathan, Thiyagaraj, Mayberry, Kalin D., Yao, Yu, Lillo Osuna, Maria Angeles, Jang, Yoonjeong, Liyanage, Janaka S.S., Blanc, Lionel, Ellis, Steven R., Wlodarski, Marcin W., Weiss, Mitchell J.
Publicado 2023

Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice

por Everette, Kelcee A., Newby, Gregory A., Levine, Rachel M., Mayberry, Kalin, Jang, Yoonjeong, Mayuranathan, Thiyagaraj, Nimmagadda, Nikitha, Dempsey, Erin, Li, Yichao, Bhoopalan, Senthil Velan, Liu, Xiong, Davis, Jessie R., Nelson, Andrew T., Chen, Peter J., Sousa, Alexander A., Cheng, Yong, Tisdale, John F., Weiss, Mitchell J., Yen, Jonathan S., Liu, David R.
Publicado 2023