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

Clinical base editing achieves transfusion independence and durable fetal hemoglobin expression in diverse β-hemoglobinopathy patients, including an African SCD case, with no adverse effects.

• Why it matters: Addressing the limited applicability of previous gene editing approaches across different genetic backgrounds 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 common regional mutations, applying transformer base editing (tBE) and monitoring outcomes over 12 months.
• The result: All patients experienced hematopoietic recovery, discontinued transfusions, and maintained high-level HbF expression without off-target effects or adverse events, supporting broader use of tBE for diverse populations.