Highly efficient therapeutic gene editing of human hematopoietic stem cells.

Volume: 25, Issue: 5, Pages: 776 - 783
Published: Mar 25, 2019
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
Volume
25
Issue
5
Pages
776 - 783
Citation AnalysisPro
  • Scinapse’s Top 10 Citation Journals & Affiliations graph reveals the quality and authenticity of citations received by a paper.
  • Discover whether citations have been inflated due to self-citations, or if citations include institutional bias.