Author

Zhen-Wei Wei

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Sep 2026

Clinical base editing for β-hemoglobinopathies across different genetic backgrounds.

β-Hemoglobinopathies are monogenic disorders. We previously applied a transformer base editor (tBE) to reactivate fetal hemoglobin (HbF) expression, and five Chinese transfusion-dependent β-thalassemia (TDT) patients achieved transfusion independence. However, the applicability of tBE to sickle cell disease (SCD) and g...

Rong-Rong Liu, Yong-Rong Lai, Li-Jie Wang et al. · Cell Stem Cell · 0 citations

Open access Sep 2026

Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase IV, open-label, single-centre trial.

We report the results of an open-label, single-centre phase 4 clinical trial evaluating haploidentical haematopoietic stem cell transplantation (Haplo-HSCT) in patients with transfusion-dependent thalassaemia (TDT). A total of 134 patients with TDT were enrolled; the median age was 8.7 years (interquartile range [IQR],...

Xiao-Xuan Lai, Rong-Rong Liu, Hongwen Xiao et al. · British Journal of Haematolo... · 0 citations