Biblio
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.
Enhanced Cas12a editing in mammalian cells and zebrafish. Nucleic Acids Res. 2019.
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv. 2019;3(21):3379-3392.