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

Title
Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells
Authors
Keywords
genome editing, gene therapy, BCL11A, hemoglobinopathies, fetal hemoglobin, fetal globin reactivation, zinc finger nucleases
Journal
Publisher
Elsevier BV
Online
2017-01-11
DOI
10.1016/j.omtm.2016.12.009

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