γ-globin reactivated autologous hematopoietic stem cells

γ-globin reactivated autologous hematopoietic stem cells is a gene therapy approach for treating hemoglobinopathies such as sickle cell disease (SCD) and β-thalassemia. This therapy involves collecting a patient's own hematopoietic stem cells (HSCs), genetically modifying them ex vivo using CRISPR-Cas9 gene editing technology to reactivate fetal hemoglobin (HbF) production, and then reinfusing these modified cells back into the patient after myeloablative conditioning. The genetic modification typically targets the promoter regions of γ-globin genes (HBG1/HBG2) to disrupt the binding site of BCL11A, a transcriptional repressor of fetal hemoglobin. This leads to increased production of fetal hemoglobin, which can compensate for defective adult hemoglobin in patients with these disorders.