Base editing of β0-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies
Sci Transl Med. 2025 Nov 26;17(826):eadt8617. doi: 10.1126/scitranslmed.adt8617. Epub 2025 Nov 26. ABSTRACT Gene therapy has emerged as a promising curative treatment for β-hemoglobinopathies, the …