Article

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

2020-10-28

Abstract excerpt

<h4>ABSTRACT</h4> β-thalassemia pathology is not only due to loss of β-globin ( HBB ), but also erythrotoxic accumulation and aggregation of the β-globin binding partner, α-globin ( HBA1/2 ). Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace the entire HBA1 gene with a full-length HBB transgene in β-thalassemia-derived hematopoietic stem and progenitor cells (HSPCs), which is suff...

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Literature Corpus work: 5e3cda10-3787-5295-850a-e5e27108a8ba
DOI: 10.1101/2020.10.28.359315

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