Clinical application of base editing for treating β-thalassaemia - PubMed
3 hours ago
- #gene editing
- #β-thalassaemia
- #clinical trial
- Phase 1 trial shows transformer base editor CS-101 reactivates fetal hemoglobin in β-thalassaemia patients.
- Five patients achieved transfusion independence with sustained hemoglobin and fetal hemoglobin levels after treatment.
- No deaths or cancers reported; adverse events aligned with standard transplantation procedures.