Flipping the fetal switch across β-hemoglobinopathies.

Transformer base editing of HBG1/2 promoters was successfully applied across diverse β-hemoglobinopathies, including sickle cell disease and various β-thalassemia genotypes, in four patients.

• Why it matters: Addressing the limited scope of previous gene editing approaches, this work explores broader applicability to different β-hemoglobinopathy conditions, emphasizing the need for scalable and accessible cures.
• What they did: Liu et al. extended the use of transformer base editing from transfusion-dependent β-thalassemia to one sickle cell disease patient and three β-thalassemia patients with diverse genotypes, demonstrating meaningful responses.
• The result: The findings suggest potential for wider clinical translation, but challenges related to scale, genomic surveillance, treatment burden, and access remain critical for future development.