Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells

Gene editing the BCL11A erythroid enhancer is a validated approach to fetal hemoglobin (HbF) induction for β-hemoglobinopathy therapy, though heterogeneity in edit allele distribution and HbF response may impact its safety and efficacy. Here, we compare combined CRISPR-Cas9 editing of the BCL11A +58...

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Veröffentlicht in:	Cell stem cell 2024-12
Hauptverfasser:	Zeng, Jing, Nguyen, My Anh, Liu, Pengpeng, da Silva, Lucas Ferreira, Levesque, Sébastien, Lin, Linda Y., Justus, David G., Petri, Karl, Clement, Kendell, Porter, Shaina N., Verma, Archana, Neri, Nola R., Rosanwo, Tolulope, Ciuculescu, Marioara-Felicia, Abriss, Daniela, Mintzer, Esther, Maitland, Stacy A., Demirci, Selami, Cha, Hye Ji, Orkin, Stuart H., Tisdale, John F., Williams, David A., Zhu, Lihua Julie, Pruett-Miller, Shondra M., Pinello, Luca, Joung, J. Keith, Pattanayak, Vikram, Manis, John P., Armant, Myriam, Pellin, Danilo, Brendel, Christian, Wolfe, Scot A., Bauer, Daniel E.
Format:	Artikel
Sprache:	eng
Schlagworte:	BCL11A enhancers fetal hemoglobin genotoxicity hematopoietic stem cells sickle cell disease therapeutic gene editing
Online-Zugang:	Volltext
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