Biblio
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice. Nat Biomed Eng. 2023.
Outcomes and long-term effects of hematopoietic stem cell transplant in sickle cell disease. Expert Rev Hematol. 2023.
. Potent and uniform fetal hemoglobin induction via base editing. Nat Genet. 2023.
Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease. Blood Adv. 2021;5(9):2403-2411.
. Hematopoietic-Stem-Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies. Cell Stem Cell. 2021;28(2):191-208.
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2019 sickle cell disease guidelines by the American Society of Hematology: methodology, challenges, and innovations. Blood Adv. 2019;3(23):3945-3950.
CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges. Adv Exp Med Biol. 2019.
. Fetal hemoglobin (HbF) and F-cell variance in mobilized CD34 cell transplanted rhesus monkeys. Exp Hematol. 2019.
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv. 2019;3(21):3379-3392.
Total body irradiation must be delivered at high dose for efficient engraftment and tolerance in a rhesus stem cell gene therapy model. Mol Ther Methods Clin Dev. 2016;3:16059.
. Decitabine Suspends Human CD34+ Cell Differentiation and Proliferation during Lentiviral Transduction. PLoS One. 2014;9(8):e104022.
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