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Received — 13 April 2026 ⏭

Nature - Issue - nature.com science feeds
Clinical application of base editing for treating β-thalassaemia Yongrong Lai · Rongrong Liu · Lijie Wang · Xu-Kai Ma · Yaliang Li · Gaohui Yang · Lingling Shi · Yi-Lin Guo · Zhenbin Wei · Xuemei Zhou · Wenchao Xu · Yaofeng Hou · Annarita Miccio · Bei Yang · Xiaodun Mou · Li Yang · Jia Chen
Nature, Published online: 08 April 2026; doi:10.1038/s41586-026-10342-9A clinical phase 1 trial of a single infusion of CS-101, CD34+ cells modified using a transformer base editor to reactivate fetal haemoglobin production, led to early and enduring transfusion independence in patients with β-thalassaemia.
8 April 2026 at 08:00

Clinical application of base editing for treating β-thalassaemia

Nature - Issue - nature.com science feeds

By: Yongrong Lai · Rongrong Liu · Lijie Wang · Xu-Kai Ma · Yaliang Li · Gaohui Yang · Lingling Shi · Yi-Lin Guo · Zhenbin Wei · Xuemei Zhou · Wenchao Xu · Yaofeng Hou · Annarita Miccio · Bei Yang · Xiaodun Mou · Li Yang · Jia Chen

8 April 2026 at 08:00

Nature, Published online: 08 April 2026; doi:10.1038/s41586-026-10342-9

A clinical phase 1 trial of a single infusion of CS-101, CD34+ cells modified using a transformer base editor to reactivate fetal haemoglobin production, led to early and enduring transfusion independence in patients with β-thalassaemia.