BCL11A +58/+55 Enhancer-Editing Facilitates HSPC Engraftment and HbF Induction in Rhesus Macaques Conditioned with a CD45 Antibody-Drug Conjugate

Selami Demirci,Jing Zeng,Rahul Palchaudhuri,Chuanfeng Wu,Diana M. Abraham, Taha B. Hayal,Khaled Essawi,My Anh Nguyen,Ulana Stasula,Rebecca Chu,Alexis Leonard,Shaina N. Porter, Muhammad Behroz Naeem Khan, Gabriela Hinojosa,Naoya Uchida,Sogun Hong,Cicera R. Lazzarotto,Nola R. Neri, Lucas Ferreira da Silva,Danilo Pellin

Cell Stem Cell（2024）

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fetal hemoglobin,γ-globin,sickle cell disease,therapeutic gene editing,antibody conditioning,busulfan,HSC transplantation,β-globin disorders,hydroxyurea,CRISPR-Cas9

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