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Article

Therapeutic adenine base editing of human hematopoietic stem cells

13 Jan 2023

Abstract excerpt

Abstract In β-thalassemia, either γ-globin induction to form fetal hemoglobin (α2γ2) or β-globin repair to restore adult hemoglobin (α2β2) could be therapeutic. ABE8e, a recently evolved adenine base editor variant, can achieve efficient adenine conversion, yet its application in patient-derived hematopoietic stem cells needs further exploration. Here, we purified ABE8e for ribonucleoprotein electroporation of...

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CRISPR and Genetic Engineering
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 36639729
DOI: 10.1038/s41467-022-35508-7

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