Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Abstract
Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by induction of fetal hemoglobin (HbF, α2γ2)1. Previously, we and others have shown that core sequences at the BCL11A erythroid enhancer are required for repression of HbF in adult-stage erythroid cells but are dispensable in non-erythroid cells2–6. CRISPR–Cas9-mediated gene...
Paper Details
Title
Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Published Date
Mar 25, 2019
Journal
Volume
25
Issue
5
Pages
776 - 783
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Notes
History