关键词:  , β-血红蛋白病；基因组编辑；胎儿血红蛋白

Abstract: β-hemoglobinopathy is the most common genetic disease in the world. The initial treatment strategy is viral vector-mediated gene therapy. Due to the high cost, limited efficacy and potential safety problems, the progress is slow. In recent years, genome editing technology has been a powerful tool for the development of new cures for β-hemoglobinopathy. This article reviewed the latest developments in fetal hemoglobin regeneration strategies for the treatment of β-hemoglobinopathy. The stratergy of disrupting the BCL11A erythroid enhancer is favored due to its safety, effectiveness and higher clinical value. Looking forward to development of gene therapy strategy for β-hemoglobinopathy, it is expected to completely cure the two inherited blood diseases of β-thalassemia and sickle cell anemia.