Clinical application of base editing for treating β-thalassaemia

www.nature.com

Clinical application of base editing for treating β-thalassaemia

www.nature.com

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NatureEnglish · 13 hours ago

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.

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