Betibeglogene autotemcel gene therapy for non–β<sup>0</sup>/β<sup>0</sup> genotype β-thalassemia

BACKGROUND Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with the BB305 lentiviral vector encoding the β-globin (βA-T87Q) gene. METHODS In this open-label, phase 3 study, we...

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Bibliographic Details
Main Author: Locatelli F.
Other Authors: Mahidol University
Format: Article
Published: 2023
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Online Access:https://repository.li.mahidol.ac.th/handle/123456789/86120
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Institution: Mahidol University
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