Page 212 - 2021_10-Haematologica-web

LETTERS TO THE EDITOR
Inclusion of a short hairpin RNA targeting BCL11A into a b-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin
Addition of a functional copy of the b-globin gene and reactivation of fetal hemoglobin (HbF) are promising therapeutic approaches for b-globinopathies such as sick- le cell disease (SCD) and b-thalassemia. Results from ongoing clinical trials for b-globinopathies indicate that a
successful outcome is genotype-dependent. In studies that included patients with the b0/b0 genotype or SCD, vectors expressing a single curative gene have failed or have required several integrations per genome to produce enough adult hemoglobin (HbA) levels to correct the phenotype of the patients.1-6 Therefore, to achieve HbA synthesis at therapeutic levels in the most severe of geno- types, and with minimal vector copy number (VCN) per cell, more powerful and versatile vectors are required.
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Figure 1. Legend on following page.
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haematologica | 2021; 106(10)