Identification of Small Molecules That Induce Therapeutic Levels of Fetal Hemoglobin for Treatment of Sickle Cell Disease By Pairing Machine Learning with High-Resolution Single Cell RNA Sequencing Maps of Adult and Fetal Human Erythropoiesis

Induction of fetal hemoglobin (HbF) to treat sickle cell disease (SCD) and beta-thalassemia has been validated as a clinical strategy to ameliorate these diseases. However, despite advances in cell and gene therapy to treat SCD, current approaches still require myeloablation and bone marrow transpla...

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Veröffentlicht in:	Blood 2021-11, Vol.138 (Supplement 1), p.2022-2022
Hauptverfasser:	Cortes, Mauricio, Monti, Anthony J, Sun, Sunny, Lynch, Orna, Xia, Youli, Lin, Sarah, Malamas, Michael, Steelman, Scott, Krishnamoorthy, Sriram, Stewart, Morag, Tozzo, Effie
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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