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Reference Patent

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  • Search Condition : Filter (MeSH = beta-Thalassemia* / therapy)
Species Resource RRC ID Title Journal Published Link
Human and Animal Cells HUDEP-2(RCB4557) 77350 Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype. Sci Adv 2020-2-1 Pubmed Full text
Human and Animal Cells HUDEP-2(RCB4557) 77259 Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells. Blood Adv 2021-3-9 Pubmed Full text
Human and Animal Cells HUDEP-2(RCB4557) 77239 Prime Editor 3 Mediated Beta-Thalassemia Mutations of the HBB Gene in Human Erythroid Progenitor Cells. Int J Mol Sci 2022-4-30 Pubmed Full text
Human and Animal Cells HUDEP-2(RCB4557) 77232 Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression. Blood 2022-4-7 Pubmed Full text
Human and Animal Cells HUDEP-2(RCB4557) 73033 Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression. Nat Commun 2022-11-4 Pubmed Full text
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