Biblio
An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities. Nat Biotechnol. 2018.
. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv. 2019;3(21):3379-3392.
BCL11A enhancer edited hematopoietic stem cells persist in rhesus monkeys without toxicity. J Clin Invest. 2020.
Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. Cell Stem Cell. 2020.
Dissecting ELANE neutropenia pathogenicity by human HSC gene editing. Cell Stem Cell. 2021.
Base Editing of Human Hematopoietic Stem Cells. Methods Mol Biol. 2023;2606:43-62.
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