Xiu-Li Gong

Precision base-editing of the cryptic 3’ acceptor site to correct the RNA splicing defect of β654-thalassemia

β-Thalassemia is one of the most common inherited disorders worldwide and is caused by mutations affecting β-globin production. β654 mutation (IVS2-654, C > T) is one of the most frequently occurring β-thalassemia alleles in Han Chinese population, which activates a cryptic 3′ splice site and leads to aberrant RNA spli...