Flipping the fetal switch across β-hemoglobinopathies.

Transformer base editing of HBG1/2 promoters shows promising responses across diverse β-hemoglobinopathies, including sickle cell disease and various β-thalassemia genotypes.

• Why it matters: Addressing the global burden of β-hemoglobinopathies requires effective, scalable gene editing therapies that can be broadly applied to different genetic backgrounds and patient populations.
• What they did: Liu et al. applied transformer base editing to HBG1/2 promoters in one sickle cell patient and three β-thalassemia patients with diverse genotypes, evaluating its therapeutic potential.
• The result: The meaningful responses observed suggest potential for clinical translation, but challenges remain in scaling, genomic monitoring, treatment burden, and ensuring equitable access.