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Li-Jie Wang

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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. · 0 citations

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