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Therapeutic adenine base editing of human hematopoietic stem cells

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 hematopoieti...

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Detalles Bibliográficos
Autores principales:	Liao, Jiaoyang, Chen, Shuanghong, Hsiao, Shenlin, Jiang, Yanhong, Yang, Yang, Zhang, Yuanjin, Wang, Xin, Lai, Yongrong, Bauer, Daniel E., Wu, Yuxuan
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9839747/ https://www.ncbi.nlm.nih.gov/pubmed/36639729 http://dx.doi.org/10.1038/s41467-022-35508-7

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