Healing sickle cell anemia and β-thalassemia

Abstract

Sickle cell anemia and β-thalassemia are diseases caused by point mutations in the hemoglobin β gene. Both diseases significantly affect the health of patients and are widespread worldwide. Using CRISPR-Cas technology, the fetal hemoglobin F gene was reactivated in CD34+ stem cells, allowing this form of hemoglobin to replace the mutated β variant. The cells were harvested for this procedure and later returned to the patient so that the patient’s germline was not affected. All treated patients produced a health-relevant amount of hemoglobin F and showed significantly reduced or no symptoms at all of the disease. The EU, the UK and the USA have already approved this first CRISPR-Cas therapy.