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References from Flipping the fetal switch across β-hemoglobinopathies. Local targets link to admitted publications; unresolved targets remain external evidence.
Clinical base editing for β-hemoglobinopathies across different genetic backgrounds
10.1016/j.stem.2026.08.009 · 2026 · External reference
Clinical application of base editing for treating β-thalassaemia
10.1038/s41586-026-10342-9 · 2026 · External reference
Biologic and clinical efficacy of LentiGlobin for sickle cell disease
10.1056/nejmoa2117175 · 2022 · External reference
Gene therapy in patients with transfusion-dependent β-thalassemia
10.1056/nejmoa1705342 · 2018 · External reference
Exa-cel in children with transfusion-dependent β-thalassemia or sickle cell disease
10.1056/nejmoa2603387 · 2026 · External reference
CRISPR-Cas9 editing of the HBG1 and HBG2 promoters to treat sickle cell disease
10.1056/nejmoa2215643 · 2023 · External reference
CRISPR-Cas12a gene editing of HBG1 and HBG2 promoters to treat sickle cell disease
10.1056/nejmoa2415550 · 2026 · External reference
Base editing of HBG1 and HBG2 promoters for sickle cell disease
10.1056/nejmoa2504835 · 2026 · External reference
How I treat sickle cell disease with gene therapy
10.1182/blood.2024024519 · 2024 · External reference
Dismantling cost and infrastructure barriers to equitable access to gene therapies for sickle cell disease
10.1016/s2352-3026(24)00175-3 · 2024 · External reference
