Genetic disruption of the KLF1 gene to overexpress the γ‐globin gene using the CRISPR/Cas9 system

Abstract

β‐thalassemia comprises a major group of human genetic disorders involving a decrease in or an end to the normal synthesis of the β‐globin chains of hemoglobin. KLF1 is a key regulatory molecule involved in the γ‐ to β‐globin gene switching process directly inducing the expression of the β‐globin gene and indirectly repressing γ‐globin. The present study aimed to investigate the ability of an engineered CRISPR/Cas9 system with respect to disrupting the KLF1 gene to inhibit the γ‐ to β‐hemoglobin switching process in K562 cells.