CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Abstract
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin in erythroid cells. We performed electroporation of CD34+ hematopoietic stem and progenitor cells obtained from healthy donors, with CRISPR-Cas9 targeting the BCL11A erythroid-specific enhancer....
Paper Details
Title
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Published Date
Jan 21, 2021
Volume
384
Issue
3
Pages
252 - 260
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