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

Base editing treatment achieved transfusion independence and durable fetal hemoglobin expression in four patients with diverse β-hemoglobinopathies, including an African SCD patient.

• Why it matters: Addressing the applicability of base editing across different genetic backgrounds and disease types is crucial for expanding treatment options for β-hemoglobinopathies worldwide.
• What they did: Researchers conducted three clinical trials involving one African SCD patient and three TDT patients with mutations common in South and Southeast Asia, monitoring hematopoietic recovery and long-term outcomes.
• The result: All patients experienced transfusion independence, sustained high-level HbF expression, and no adverse off-target effects or vaso-occlusive episodes, supporting broader use of tBE for diverse populations.