Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies
Dear Editor,
Mutations in the β-globin gene,the essential component of adult hemoglobin (HbA;α2β2),results in either a production of aberrant sickle hemoglobin (HbS) leading to sickle cell disease (SCD) or an insufficient β-globin synthesis leading to β-thalassemia.These two major forms of β-hemoglobinopathies cause impaired erythropoiesis and life-threatening anemia.Clinical evidence has suggested that reactivation of fetal y-globin (HBG) gene expression which is normally silenced after birth by certain genetic mutations can ameliorate the clinical course of β-hemoglobinopathies.1,2 In β-thalassemia,elevated levels of fetal y-globin interact with α-globin to form fetal hemoglobin (HbF;α2γ2) restoring the α/β-like globin ratio and in SCD the y-globin reduces HbS polymerization.
30
2020-06-15（万方平台首次上网日期，不代表论文的发表时间）
共3页
276-278
