Therapeutic adenine base editing of human hematopoietic stem cells
Therapeutic adenine base editing of human hematopoietic stem cells
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London: Nature Publishing Group UK
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English
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London: Nature Publishing Group UK
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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 p...
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Therapeutic adenine base editing of human hematopoietic stem cells
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TN_cdi_doaj_primary_oai_doaj_org_article_d4f2094ecf5e4ebe9d711d75133738a7
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_d4f2094ecf5e4ebe9d711d75133738a7
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2041-1723
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2041-1723
DOI
10.1038/s41467-022-35508-7