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.
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