Article

Gene therapy using optimized LentiHBB<sup>T87Q</sup>vector in two patients with transfusion dependent β-thalassemia

2023-03-27

Abstract excerpt

<h4>Background</h4> Gene therapy is gradually becoming recognized as a possibly curative therapeutic strategy for transfusion-dependent β-thalassemia (TDT). Gene therapy addresses the problem of donor scarcity through the application of autologous hematopoietic stem cells (HSCs), which also can reduce the risks that accompany allogeneic HSC transplantation. When using gene addition strategy, lentiviral vector is c...

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Literature Corpus work: 4638b103-009b-5c7f-9c96-3da3285b9cf2
DOI: 10.1101/2023.03.21.23287513

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