Biblio
Base Editing of Human Hematopoietic Stem Cells. Methods Mol Biol. 2023;2606:43-62.
. Dissecting ELANE neutropenia pathogenicity by human HSC gene editing. Cell Stem Cell. 2021.
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.
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv. 2019;3(21):3379-3392.
An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities. Nat Biotechnol. 2018.
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