Induction of HbF by a genome editing-based deletion can ameliorate SCD. This illustration depicts the human β-globin locus on chromosome 11 (chr11) with a 3.5-kb silencer region upstream of the δ-globin gene. Typical deletions implicated in HPFH and δβ-thalassemia, as well as the Corfu thalassemia deletion, are illustrated below the locus. The schematic shows that disruption of the silencer region, in addition to the δ- and β-globin genes, using genome editing tools (depicted as scissors) can lead to a robust elevation in HbF production and ameliorate the SCD phenotype. HSs, hypersensitivity sites; 3′HS1, downstream hypersensitivity site; LCR, locus control region. Professional illustration by Patrick Lane, ScEYEnce Studios.

Induction of HbF by a genome editing-based deletion can ameliorate SCD. This illustration depicts the human β-globin locus on chromosome 11 (chr11) with a 3.5-kb silencer region upstream of the δ-globin gene. Typical deletions implicated in HPFH and δβ-thalassemia, as well as the Corfu thalassemia deletion, are illustrated below the locus. The schematic shows that disruption of the silencer region, in addition to the δ- and β-globin genes, using genome editing tools (depicted as scissors) can lead to a robust elevation in HbF production and ameliorate the SCD phenotype. HSs, hypersensitivity sites; 3′HS1, downstream hypersensitivity site; LCR, locus control region. Professional illustration by Patrick Lane, ScEYEnce Studios.

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