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

摘要

Background-thalassemia comprises a major group of human genetic disorders involving a decrease in or an end to the normal synthesis of the -globin chains of hemoglobin. KLF1 is a key regulatory molecule involved in the - to -globin gene switching process directly inducing the expression of the -globin gene and indirectly repressing -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 - to -hemoglobin switching process in K562 cells.