Adenine Base Editing for Sickle Cell Disease
September 12, 2023 | Terry Sharrer
“Base editing—the genome editing technique that alters base pairs using a nuclease-deficient Cas9 fused to a deaminase—was found to restart fetal hemoglobin expression in sickle cell disease (SCD) patient cells. This result stems from work that compared five genome editing strategies, in CD34+ hematopoietic stem and progenitor cells, using either Cas9 nuclease or adenine base editors. The findings suggest that adenosine base editing raised the expression of fetal hemoglobin to higher, more stable, and more uniform levels than other genome editing technologies that use CRISPR/Cas9 nuclease in human hematopoietic stem cells.” MORE
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