Sep 2026
Clinical, immunological, and molecular characteristics of severe combined immune deficiency in China
Fei-Fan Xiao, Qin-Hua Zhou, Cheng-Hao Wang et al.
· Human Genetics · 0 citations
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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