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Fetal hemoglobin regeneration for therapy of β-hemoglobinopathies

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  • 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 date: 2021-11-12

  Online published: 2022-07-18

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. 

Cite this article

ZHAO Feiyan, WU Yuxuan . Fetal hemoglobin regeneration for therapy of β-hemoglobinopathies[J]. Chinese Journal of Nature, 2022 , 44(5) : 389 -397 . DOI: 10.3969/j.issn.0253-9608.2022.03.011

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