Biblio
2019 sickle cell disease guidelines by the American Society of Hematology: methodology, challenges, and innovations. Blood Adv. 2019;3(23):3945-3950.
BCL11A enhancer edited hematopoietic stem cells persist in rhesus monkeys without toxicity. J Clin Invest. 2020.
Biallelic correction of sickle cell disease-derived iPSCs confirmed at the protein level through serum-free iPS-sac/erythroid differentiation. Stem Cells Transl Med. 2020.
. CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges. Adv Exp Med Biol. 2019.
. Cytomegalovirus infection incidence and risk factors across diverse hematopoietic cell transplantation platforms using a standardized monitoring and treatment approach: a comprehensive evaluation from a single institution. Biol Blood Marrow Transplant. 2018.
Decitabine Suspends Human CD34+ Cell Differentiation and Proliferation during Lentiviral Transduction. PLoS One. 2014;9(8):e104022.
. Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease. Blood Adv. 2021;5(9):2403-2411.
Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models. Cytotherapy. 2024.
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice. Nat Biomed Eng. 2023.
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.
Hematopoietic-Stem-Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies. Cell Stem Cell. 2021;28(2):191-208.
. Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype. JAMA. 2014;312(1):48-56.
. Non-myeloablative human leukocyte antigen-matched related donor transplantation in sickle cell disease: outcomes from three independent centres. Br J Haematol. 2021.
. 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.
Robust generation of erythroid and multilineage hematopoietic progenitors from human iPSCs using a scalable monolayer culture system. Stem Cell Res. 2019;41:101600.
Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells. Cell Rep Methods. 2023;3(4):100460.
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.
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