Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells
Jing Zeng, My Anh Nguyen, Pengpeng Liu, Lucas Ferreira da Silva, Sébastien Levesque, Linda Yingqi Lin, David G. Justus, Karl Petri, Kendell Clement, Shaina N. Porter, Archana Verma, Nola R. Neri, Tolulope O. Rosanwo, Marioara-Felicia Ciuculescu, Daniela Abriss, , Daniel E. Bauer
This study shows that gene editing of HSCs without ex vivo culture reduces genotoxicity while maintaining therapeutic efficacy for beta-hemoglobinopathy treatment.
Cell stem cell · Dec 12, 2024Peer-reviewedGenetics