Chinese Journal of Nature ›› 2022, Vol. 44 ›› Issue (5): 389-397.doi: 10.3969/j.issn.0253-9608.2022.03.011

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Fetal hemoglobin regeneration for therapy of β-hemoglobinopathies

ZHAO Feiyan, WU Yuxuan   

  1. Shanghai Key Laboratory of Regulatory Biology, Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, School of Life Sciences, East China Normal University, Shanghai 200241, China
  • Received:2021-11-12 Online:2022-10-25 Published:2022-10-25

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.