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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| 格式: | Article |
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2023
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| 在線閱讀: | https://repository.li.mahidol.ac.th/handle/123456789/86120 |
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| 機構: | Mahidol University |