Biblio
Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin. Curr Opin Genet Dev. 2015;33:62-70.
. Analyzing CRISPR genome-editing experiments with CRISPResso. Nat Biotechnol. 2016;34(7):695-697.
. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016.
Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin. Science. 2016;351(6270):285-289.
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.
Rational targeting of a NuRD subcomplex guided by comprehensive in situ mutagenesis. Nat Genet. 2019.
Synthetic Lethality of Wnt Pathway Activation and Asparaginase in Drug-Resistant Acute Leukemias. Cancer Cell. 2019;35(4):664-676.e7.
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.
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DNAJB1-PRKACA in HEK293T cells induces LINC00473 overexpression that depends on PKA signaling. PLoS One. 2022;17(2):e0263829.
Pervasive donor DNA integration defies precision gene editing of hematopoietic stem cells. Cell Stem Cell. 2022;29(10):1426-1427.
. Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing. Nat Commun. 2023;14(1):212.
. Base Editing of Human Hematopoietic Stem Cells. Methods Mol Biol. 2023;2606:43-62.
. Gene correction for sickle cell disease hits its prime. Nat Biomed Eng. 2023;7(5):605-606.
. Molecular Basis and Genetic Modifiers of Thalassemia. Hematol Oncol Clin North Am. 2023;37(2):273-299.
. RUNX1 mutations mitigate quiescence to promote transformation of hematopoietic progenitors in Fanconi anemia. Leukemia. 2023.
Therapeutic adenine base editing of human hematopoietic stem cells. Nat Commun. 2023;14(1):207.
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