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Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells

11 Jan 2017

Abstract excerpt

To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or at the GATAA motif in the intronic erythroid-specific enhancer, using zinc finger nucleases in human bone marrow (BM) CD34 + hematopoietic stem and progenitor cells (HSPCs). Both targeting strategies upregulated fetal globin...

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PMID: 28344999
DOI: 10.1016/j.omtm.2016.12.009

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