Selami Demirci, Muhammad B N Khan, Gabriela Hinojosa, Anh Le, Alexis Leonard, Khaled Essawi, Bjorg Gudmundsdottir, Xiong Liu, Jing Zeng, Zaina Inam, Rebecca Chu, Naoya Uchida, Daisuke Araki, Evan London, Henna Butt, Stacy A Maitland, Daniel E Bauer, Scot A Wolfe, Andre Larochelle, John F Tisdale
Ex vivo resting culture is a standard procedure following genome editing in hematopoietic stem and progenitor cells (HSPCs). However, prolonged culture may critically affect cell viability and stem cell function. We investigated whether varying durations of culture resting times impact the engraftment efficiency of human CD34+ HSPCs edited at the BCL11A enhancer, a key regulator in the expression of fetal hemoglobin. We employed electroporation to introduce CRISPR-Cas9 components for BCL11A enhancer editing and compared outcomes with nonelectroporated (NEP) and electroporated-only (EP) control groups...
February 24, 2024: Cytotherapy