WeatherallDJ. Phenotype-genotype relationships in monogenic disease: lessons from the thalassaemias. Nat Rev Genet, 2001; 2:245–255.
2.
OuyangW, DongG, ZhaoW, et al.Restoration of β-globin expression with optimally designed lentiviral vector for β-thalassemia treatment in Chinese patients. Hum Gene Ther, 2021; 32:481–494.
3.
ThompsonAA, WaltersMC, KwiatkowskiJ, et al.Gene therapy in patients with transfusion-dependent beta-thalassemia. N Engl J Med, 2018; 378:1479–1493.