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5 Hits
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